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eventually fatal.
SRP-9005 (LGMD2C Î-SARCOGLYCAN) SRP-9005 (LGMD2C Îł-sarcoglycan) | Sarepta Therapeutics. Main navigation. Our Disease Areas. Duchenne Muscular Dystrophy. Limb-girdle Muscular Dystrophy. Mucopolysaccharidosis Type IIIA. Charcot-Marie-Tooth Disease. Our Science. Gene Therapy Engine.INVESTOR RELATIONS
Investor Relations. At Sarepta, we are leading a revolution in precision genetic medicine and every day is an opportunity to change the lives of people living with rare disease. The Company has built an impressive position in Duchenne muscular dystrophy (DMD) and in gene therapies for limb-girdle muscular dystrophy diseases (LGMD), Charcot SAREPTA THERAPEUTICS PROVIDES PROGRAM UPDATE FOR SRP-9001 CAMBRIDGE, Mass., Sept. 09, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that it has completed a Type C âwritten response onlyâ meeting with the Office of Tissues and Advanced Therapies (OTAT), part of the Center for Biologics Evaluation and Research (CBER) at the U.S. Food and Drug SAREPTA THERAPEUTICS ANNOUNCES FDA ACCEPTANCE OF This section of our website may contain dated or archived information which should not be considered current and may no longer be accurate. For current information, you are encouraged to review our most recent official corporate documents on file with the U.S. Securities and Exchange Commission.PRESS RELEASES
Sarepta Therapeutics Announces Inducement Grants Under Nasdaq Listing Rule 5635 (c) (4) 5/18/21. Sarepta Therapeuticsâ Investigational Gene Therapy for the Treatment of Duchenne Muscular Dystrophy, SRP-9001, Demonstrates Robust Expression and Consistent Safety Profile Using Sareptaâs Commercial Process Material. 5/17/21. CLINICAL UPDATE: STUDY SRP-9001-103: 12-WEEK EXPRESSION « Back. Clinical Update: Study SRP-9001-103: 12-Week Expression and Safety Data Using Commercially Representative Material. 05/18/21 at8:30 AM EDT
SAREPTA EXERCISES OPTION TO ACQUIRE MYONEXUS THERAPEUTICS CAMBRIDGE, Mass., Feb. 27, 2019 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, announced today that it has exercised its option to acquire Myonexus Therapeutics, a clinical-stage biotechnology company developing transformative gene therapies forfive LGMDs: LGMD2E
SAREPTA THERAPEUTICS We see a revolution. Sarepta is a global biotechnology company on an urgent mission: engineer precision genetic medicine to reclaim futures otherwise impacted or cut short by rare diseases. Weâre ushering in a new era of drug development, with the goal of shortening the time from lab to patient, building the worldâs largest gene therapy NEWS & PRESS RELEASES Read news and articles from Sarepta, a global biotechnology company developing potentially life-changing precision genetic medicine. DUCHENNE MUSCULAR DYSTROPHY (DMD) Duchenne muscular dystrophy, sometimes shortened to DMD or just Duchenne, is a rare genetic disease. It predominantly affects males, but, in rare cases, can also affect females. Duchenne causes the muscles in the body to become weak and damaged over time and iseventually fatal.
SRP-9005 (LGMD2C Î-SARCOGLYCAN) SRP-9005 (LGMD2C Îł-sarcoglycan) | Sarepta Therapeutics. Main navigation. Our Disease Areas. Duchenne Muscular Dystrophy. Limb-girdle Muscular Dystrophy. Mucopolysaccharidosis Type IIIA. Charcot-Marie-Tooth Disease. Our Science. Gene Therapy Engine.INVESTOR RELATIONS
Investor Relations. At Sarepta, we are leading a revolution in precision genetic medicine and every day is an opportunity to change the lives of people living with rare disease. The Company has built an impressive position in Duchenne muscular dystrophy (DMD) and in gene therapies for limb-girdle muscular dystrophy diseases (LGMD), Charcot SAREPTA THERAPEUTICS PROVIDES PROGRAM UPDATE FOR SRP-9001 CAMBRIDGE, Mass., Sept. 09, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that it has completed a Type C âwritten response onlyâ meeting with the Office of Tissues and Advanced Therapies (OTAT), part of the Center for Biologics Evaluation and Research (CBER) at the U.S. Food and Drug SAREPTA THERAPEUTICS ANNOUNCES FDA ACCEPTANCE OF This section of our website may contain dated or archived information which should not be considered current and may no longer be accurate. For current information, you are encouraged to review our most recent official corporate documents on file with the U.S. Securities and Exchange Commission.PRESS RELEASES
Sarepta Therapeutics Announces Inducement Grants Under Nasdaq Listing Rule 5635 (c) (4) 5/18/21. Sarepta Therapeuticsâ Investigational Gene Therapy for the Treatment of Duchenne Muscular Dystrophy, SRP-9001, Demonstrates Robust Expression and Consistent Safety Profile Using Sareptaâs Commercial Process Material. 5/17/21. CLINICAL UPDATE: STUDY SRP-9001-103: 12-WEEK EXPRESSION « Back. Clinical Update: Study SRP-9001-103: 12-Week Expression and Safety Data Using Commercially Representative Material. 05/18/21 at8:30 AM EDT
SAREPTA EXERCISES OPTION TO ACQUIRE MYONEXUS THERAPEUTICS CAMBRIDGE, Mass., Feb. 27, 2019 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, announced today that it has exercised its option to acquire Myonexus Therapeutics, a clinical-stage biotechnology company developing transformative gene therapies forfive LGMDs: LGMD2E
PIPELINE & PRODUCTS
Pipeline & Products | Sarepta Therapeutics. At Sarepta, we are working with urgency to develop breakthrough therapies to treat genetic diseases. Currently, we have more than 40 investigational therapies in various stages of developmentâmany already in late-stage clinical trials. In many cases, development is being accelerated by our geneCLINICAL TRIALS
Brief Summary: This study is designed to assess the efficacy, safety, tolerability, and pharmacokinetics (PK) of AVI-4658 (eteplirsen) in both 50.0 mg/kg and 30.0 mg/kg doses administered over 24 weeks in subjects diagnosed with Duchenne muscular dystrophy GENE EDITING RESEARCH At Sarepta, we are committed to exploring every modality of precision genetic medicine that may help advance our mission to rescue lives otherwise impacted or stolen by rare disease. We believe gene editing, although early, has the potential to revolutionize the treatment of NORTH STAR AMBULATORY ASSESSMENT North Star Ambulatory Assessment âąThe NSAA is only given to patients who are ambulant, or able to walk on their own.2 âą The NSAA contains universally used methods to practically and reliably measure motor function.2 In clinical trials, the NSAA can be used as an endpoint to measure a personâs functional abilities before and after receiving an investigational therapy.1 ROUTE 79 SCHOLARSHIP PROGRAM Route 79, The Duchenne Scholarship Program, is designed to help students diagnosed with Duchenne muscular dystrophy pursue their post-high school educational goals. The scholarship is named after the79 exons in the dystrophin gene impacted by Duchenne. Sarepta knows that the route traveled by every person with Duchenne is distinct. LIMB GIRDLE MUSCULAR DYSTROPHY (LGMD) Limb-girdle muscular dystrophy (LGMD) is a group of distinct diseases that cause weakness and wasting of the muscles, generally starting with the muscles around the hips and shoulders and eventually progressing to the arms and legs. However, some subtypes start distally at the leg or arm muscles and then progress to the hip andshoulder muscles.
DOUGLAS S. INGRAM
Doug Ingram has served as President, CEO, and board member since 2017. He has no ambiguity about Sareptaâs mission: âWe know why we get up every day and work as hard as we do. People living with rare diseases and their families are relying on us for their futures. Weâre in a position to extend and change the lives of so many people. The opportunity to do good is just breathtaking.â SAREPTA THERAPEUTICSâ INVESTIGATIONAL GENE THERAPY SRP -- Protein expression in muscle was sustained for two years following treatment in the low dose cohort, with mean beta-sarcoglycan expression of 54% at 24 months, compared to 36% at Day 60, as measuredby western blot --
A SEROPREVALENCE STUDY A Seroprevalence Study EXPLORE DMD will enroll 100 individuals affected by Duchenne to see how many of these individuals havespecific types of
MOMENTUM | CLINICAL TRIALS Participant Eligibility Criteria. Your child may be eligible to participate in Momentum trial if he meets the following eligibility criteria: Ambulatory and non-ambulatory boys with DMD who are 7-21 years old (patients who enroll in Part A) or 4-21 years old (patients who enroll in Part B) Has a genetic diagnosis of DMD and anout-of-frame
SAREPTA THERAPEUTICS We see a revolution. Sarepta is a global biotechnology company on an urgent mission: engineer precision genetic medicine to reclaim futures otherwise impacted or cut short by rare diseases. Weâre ushering in a new era of drug development, with the goal of shortening the time from lab to patient, building the worldâs largest gene therapy NEWS & PRESS RELEASES Read news and articles from Sarepta, a global biotechnology company developing potentially life-changing precision genetic medicine. DUCHENNE MUSCULAR DYSTROPHY (DMD) Duchenne muscular dystrophy, sometimes shortened to DMD or just Duchenne, is a rare genetic disease. It predominantly affects males, but, in rare cases, can also affect females. Duchenne causes the muscles in the body to become weak and damaged over time and iseventually fatal.
SRP-9005 (LGMD2C Î-SARCOGLYCAN) SRP-9005 (LGMD2C Îł-sarcoglycan) | Sarepta Therapeutics. Main navigation. Our Disease Areas. Duchenne Muscular Dystrophy. Limb-girdle Muscular Dystrophy. Mucopolysaccharidosis Type IIIA. Charcot-Marie-Tooth Disease. Our Science. Gene Therapy Engine. NORTH STAR AMBULATORY ASSESSMENT North Star Ambulatory Assessment âąThe NSAA is only given to patients who are ambulant, or able to walk on their own.2 âą The NSAA contains universally used methods to practically and reliably measure motor function.2 In clinical trials, the NSAA can be used as an endpoint to measure a personâs functional abilities before and after receiving an investigational therapy.1 SAREPTA THERAPEUTICS PROVIDES PROGRAM UPDATE FOR SRP-9001 CAMBRIDGE, Mass., Sept. 09, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that it has completed a Type C âwritten response onlyâ meeting with the Office of Tissues and Advanced Therapies (OTAT), part of the Center for Biologics Evaluation and Research (CBER) at the U.S. Food and DrugPRESS RELEASES
Sarepta Therapeutics Announces Inducement Grants Under Nasdaq Listing Rule 5635 (c) (4) 5/18/21. Sarepta Therapeuticsâ Investigational Gene Therapy for the Treatment of Duchenne Muscular Dystrophy, SRP-9001, Demonstrates Robust Expression and Consistent Safety Profile Using Sareptaâs Commercial Process Material. 5/17/21. A SEROPREVALENCE STUDY A Seroprevalence Study EXPLORE DMD will enroll 100 individuals affected by Duchenne to see how many of these individuals havespecific types of
CLINICAL UPDATE: STUDY SRP-9001-103: 12-WEEK EXPRESSION « Back. Clinical Update: Study SRP-9001-103: 12-Week Expression and Safety Data Using Commercially Representative Material. 05/18/21 at8:30 AM EDT
SAREPTA EXERCISES OPTION TO ACQUIRE MYONEXUS THERAPEUTICS CAMBRIDGE, Mass., Feb. 27, 2019 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, announced today that it has exercised its option to acquire Myonexus Therapeutics, a clinical-stage biotechnology company developing transformative gene therapies forfive LGMDs: LGMD2E
SAREPTA THERAPEUTICS We see a revolution. Sarepta is a global biotechnology company on an urgent mission: engineer precision genetic medicine to reclaim futures otherwise impacted or cut short by rare diseases. Weâre ushering in a new era of drug development, with the goal of shortening the time from lab to patient, building the worldâs largest gene therapy DUCHENNE MUSCULAR DYSTROPHY (DMD) Duchenne muscular dystrophy, sometimes shortened to DMD or just Duchenne, is a rare genetic disease. It predominantly affects males, but, in rare cases, can also affect females. Duchenne causes the muscles in the body to become weak and damaged over time and iseventually fatal.
CLINICAL UPDATE: STUDY SRP-9001-103: 12-WEEK EXPRESSION « Back. Clinical Update: Study SRP-9001-103: 12-Week Expression and Safety Data Using Commercially Representative Material. 05/18/21 at8:30 AM EDT
SRP-9005 (LGMD2C Î-SARCOGLYCAN) SRP-9005 (LGMD2C Îł-sarcoglycan) | Sarepta Therapeutics. Main navigation. Our Disease Areas. Duchenne Muscular Dystrophy. Limb-girdle Muscular Dystrophy. Mucopolysaccharidosis Type IIIA. Charcot-Marie-Tooth Disease. Our Science. Gene Therapy Engine. NEWS & PRESS RELEASES Read news and articles from Sarepta, a global biotechnology company developing potentially life-changing precision genetic medicine. SAREPTA EXERCISES OPTION TO ACQUIRE MYONEXUS THERAPEUTICS CAMBRIDGE, Mass., Feb. 27, 2019 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, announced today that it has exercised its option to acquire Myonexus Therapeutics, a clinical-stage biotechnology company developing transformative gene therapies forfive LGMDs: LGMD2E
SAREPTA THERAPEUTICS PROVIDES PROGRAM UPDATE FOR SRP-9001 CAMBRIDGE, Mass., Sept. 09, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that it has completed a Type C âwritten response onlyâ meeting with the Office of Tissues and Advanced Therapies (OTAT), part of the Center for Biologics Evaluation and Research (CBER) at the U.S. Food and Drug A SEROPREVALENCE STUDY A Seroprevalence Study EXPLORE DMD will enroll 100 individuals affected by Duchenne to see how many of these individuals havespecific types of
PRESS RELEASES
Sarepta Therapeutics Announces Inducement Grants Under Nasdaq Listing Rule 5635 (c) (4) 5/18/21. Sarepta Therapeuticsâ Investigational Gene Therapy for the Treatment of Duchenne Muscular Dystrophy, SRP-9001, Demonstrates Robust Expression and Consistent Safety Profile Using Sareptaâs Commercial Process Material. 5/17/21. NORTH STAR AMBULATORY ASSESSMENT North Star Ambulatory Assessment âąThe NSAA is only given to patients who are ambulant, or able to walk on their own.2 âą The NSAA contains universally used methods to practically and reliably measure motor function.2 In clinical trials, the NSAA can be used as an endpoint to measure a personâs functional abilities before and after receiving an investigational therapy.1PIPELINE & PRODUCTS
Pipeline & Products | Sarepta Therapeutics. At Sarepta, we are working with urgency to develop breakthrough therapies to treat genetic diseases. Currently, we have more than 40 investigational therapies in various stages of developmentâmany already in late-stage clinical trials. In many cases, development is being accelerated by our gene NORTH STAR AMBULATORY ASSESSMENT North Star Ambulatory Assessment âąThe NSAA is only given to patients who are ambulant, or able to walk on their own.2 âą The NSAA contains universally used methods to practically and reliably measure motor function.2 In clinical trials, the NSAA can be used as an endpoint to measure a personâs functional abilities before and after receiving an investigational therapy.1 SAREPTA THERAPEUTICS PROVIDES PROGRAM UPDATE FOR SRP-9001 CAMBRIDGE, Mass., Sept. 09, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that it has completed a Type C âwritten response onlyâ meeting with the Office of Tissues and Advanced Therapies (OTAT), part of the Center for Biologics Evaluation and Research (CBER) at the U.S. Food and DrugCLINICAL TRIALS
Brief Summary: This study is designed to assess the efficacy, safety, tolerability, and pharmacokinetics (PK) of AVI-4658 (eteplirsen) in both 50.0 mg/kg and 30.0 mg/kg doses administered over 24 weeks in subjects diagnosed with Duchenne muscular dystrophy GENE EDITING RESEARCH At Sarepta, we are committed to exploring every modality of precision genetic medicine that may help advance our mission to rescue lives otherwise impacted or stolen by rare disease. We believe gene editing, although early, has the potential to revolutionize the treatment of ROUTE 79 SCHOLARSHIP PROGRAM Route 79, The Duchenne Scholarship Program, is designed to help students diagnosed with Duchenne muscular dystrophy pursue their post-high school educational goals. The scholarship is named after the79 exons in the dystrophin gene impacted by Duchenne. Sarepta knows that the route traveled by every person with Duchenne is distinct. A SEROPREVALENCE STUDY A Seroprevalence Study EXPLORE DMD will enroll 100 individuals affected by Duchenne to see how many of these individuals havespecific types of
SAREPTA THERAPEUTICSâ INVESTIGATIONAL GENE THERAPY SRP -- Protein expression in muscle was sustained for two years following treatment in the low dose cohort, with mean beta-sarcoglycan expression of 54% at 24 months, compared to 36% at Day 60, as measuredby western blot --
LIMB GIRDLE MUSCULAR DYSTROPHY (LGMD) Limb-girdle muscular dystrophy (LGMD) is a group of distinct diseases that cause weakness and wasting of the muscles, generally starting with the muscles around the hips and shoulders and eventually progressing to the arms and legs. However, some subtypes start distally at the leg or arm muscles and then progress to the hip andshoulder muscles.
MOMENTUM | CLINICAL TRIALS Participant Eligibility Criteria. Your child may be eligible to participate in Momentum trial if he meets the following eligibility criteria: Ambulatory and non-ambulatory boys with DMD who are 7-21 years old (patients who enroll in Part A) or 4-21 years old (patients who enroll in Part B) Has a genetic diagnosis of DMD and anout-of-frame
SAREPTA THERAPEUTICS We see a revolution. Sarepta is a global biotechnology company on an urgent mission: engineer precision genetic medicine to reclaim futures otherwise impacted or cut short by rare diseases. Weâre ushering in a new era of drug development, with the goal of shortening the time from lab to patient, building the worldâs largest gene therapy SRP-9005 (LGMD2C Î-SARCOGLYCAN) SRP-9005 (LGMD2C Îł-sarcoglycan) | Sarepta Therapeutics. Main navigation. Our Disease Areas. Duchenne Muscular Dystrophy. Limb-girdle Muscular Dystrophy. Mucopolysaccharidosis Type IIIA. Charcot-Marie-Tooth Disease. Our Science. Gene Therapy Engine. ROUTE 79 SCHOLARSHIP PROGRAM Route 79, The Duchenne Scholarship Program, is designed to help students diagnosed with Duchenne muscular dystrophy pursue their post-high school educational goals. The scholarship is named after the79 exons in the dystrophin gene impacted by Duchenne. Sarepta knows that the route traveled by every person with Duchenne is distinct. NORTH STAR AMBULATORY ASSESSMENT North Star Ambulatory Assessment âąThe NSAA is only given to patients who are ambulant, or able to walk on their own.2 âą The NSAA contains universally used methods to practically and reliably measure motor function.2 In clinical trials, the NSAA can be used as an endpoint to measure a personâs functional abilities before and after receiving an investigational therapy.1 SAREPTA THERAPEUTICS PROVIDES PROGRAM UPDATE FOR SRP-9001 CAMBRIDGE, Mass., Sept. 09, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that it has completed a Type C âwritten response onlyâ meeting with the Office of Tissues and Advanced Therapies (OTAT), part of the Center for Biologics Evaluation and Research (CBER) at the U.S. Food and DrugDOUGLAS S. INGRAM
Doug Ingram has served as President, CEO, and board member since 2017. He has no ambiguity about Sareptaâs mission: âWe know why we get up every day and work as hard as we do. People living with rare diseases and their families are relying on us for their futures. Weâre in a position to extend and change the lives of so many people. The opportunity to do good is just breathtaking.â LOUISE RODINO-KLAPAC, PHD Louise joined Sarepta in June 2018 and was appointed executive vice president, chief scientific officer in December 2020. Prior to this role Louise served as Sareptaâs senior vice president, gene therapy. She has led the design of most of Sareptaâs late-stage gene therapy candidates, has built and led Sareptaâs Gene Therapy Center of Excellence (GTCOE) in Columbus, Ohio, and hasPRE-CLINICAL
Emery-Dreifuss muscular dystrophy Type 1 (Columbia University) Read more about Emery-Dreifuss muscular dystrophy Type 1 (ColumbiaUniversity)
CLINICAL UPDATE: STUDY SRP-9001-103: 12-WEEK EXPRESSION « Back. Clinical Update: Study SRP-9001-103: 12-Week Expression and Safety Data Using Commercially Representative Material. 05/18/21 at8:30 AM EDT
SAREPTA EXERCISES OPTION TO ACQUIRE MYONEXUS THERAPEUTICS CAMBRIDGE, Mass., Feb. 27, 2019 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, announced today that it has exercised its option to acquire Myonexus Therapeutics, a clinical-stage biotechnology company developing transformative gene therapies forfive LGMDs: LGMD2E
SAREPTA THERAPEUTICS We see a revolution. Sarepta is a global biotechnology company on an urgent mission: engineer precision genetic medicine to reclaim futures otherwise impacted or cut short by rare diseases. Weâre ushering in a new era of drug development, with the goal of shortening the time from lab to patient, building the worldâs largest gene therapy SRP-9005 (LGMD2C Î-SARCOGLYCAN) SRP-9005 (LGMD2C Îł-sarcoglycan) | Sarepta Therapeutics. Main navigation. Our Disease Areas. Duchenne Muscular Dystrophy. Limb-girdle Muscular Dystrophy. Mucopolysaccharidosis Type IIIA. Charcot-Marie-Tooth Disease. Our Science. Gene Therapy Engine. ROUTE 79 SCHOLARSHIP PROGRAM Route 79, The Duchenne Scholarship Program, is designed to help students diagnosed with Duchenne muscular dystrophy pursue their post-high school educational goals. The scholarship is named after the79 exons in the dystrophin gene impacted by Duchenne. Sarepta knows that the route traveled by every person with Duchenne is distinct. NORTH STAR AMBULATORY ASSESSMENT North Star Ambulatory Assessment âąThe NSAA is only given to patients who are ambulant, or able to walk on their own.2 âą The NSAA contains universally used methods to practically and reliably measure motor function.2 In clinical trials, the NSAA can be used as an endpoint to measure a personâs functional abilities before and after receiving an investigational therapy.1 SAREPTA THERAPEUTICS PROVIDES PROGRAM UPDATE FOR SRP-9001 CAMBRIDGE, Mass., Sept. 09, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that it has completed a Type C âwritten response onlyâ meeting with the Office of Tissues and Advanced Therapies (OTAT), part of the Center for Biologics Evaluation and Research (CBER) at the U.S. Food and DrugDOUGLAS S. INGRAM
Doug Ingram has served as President, CEO, and board member since 2017. He has no ambiguity about Sareptaâs mission: âWe know why we get up every day and work as hard as we do. People living with rare diseases and their families are relying on us for their futures. Weâre in a position to extend and change the lives of so many people. The opportunity to do good is just breathtaking.â LOUISE RODINO-KLAPAC, PHD Louise joined Sarepta in June 2018 and was appointed executive vice president, chief scientific officer in December 2020. Prior to this role Louise served as Sareptaâs senior vice president, gene therapy. She has led the design of most of Sareptaâs late-stage gene therapy candidates, has built and led Sareptaâs Gene Therapy Center of Excellence (GTCOE) in Columbus, Ohio, and hasPRE-CLINICAL
Emery-Dreifuss muscular dystrophy Type 1 (Columbia University) Read more about Emery-Dreifuss muscular dystrophy Type 1 (ColumbiaUniversity)
CLINICAL UPDATE: STUDY SRP-9001-103: 12-WEEK EXPRESSION « Back. Clinical Update: Study SRP-9001-103: 12-Week Expression and Safety Data Using Commercially Representative Material. 05/18/21 at8:30 AM EDT
SAREPTA EXERCISES OPTION TO ACQUIRE MYONEXUS THERAPEUTICS CAMBRIDGE, Mass., Feb. 27, 2019 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, announced today that it has exercised its option to acquire Myonexus Therapeutics, a clinical-stage biotechnology company developing transformative gene therapies forfive LGMDs: LGMD2E
SAREPTA THERAPEUTICS PROVIDES PROGRAM UPDATE FOR SRP-9001 CAMBRIDGE, Mass., Sept. 09, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that it has completed a Type C âwritten response onlyâ meeting with the Office of Tissues and Advanced Therapies (OTAT), part of the Center for Biologics Evaluation and Research (CBER) at the U.S. Food and DrugCLINICAL TRIALS
Brief Summary: This study is designed to assess the efficacy, safety, tolerability, and pharmacokinetics (PK) of AVI-4658 (eteplirsen) in both 50.0 mg/kg and 30.0 mg/kg doses administered over 24 weeks in subjects diagnosed with Duchenne muscular dystrophyINVESTOR RELATIONS
Investor Relations. At Sarepta, we are leading a revolution in precision genetic medicine and every day is an opportunity to change the lives of people living with rare disease. The Company has built an impressive position in Duchenne muscular dystrophy (DMD) and in gene therapies for limb-girdle muscular dystrophy diseases (LGMD), CharcotDOUGLAS S. INGRAM
Doug Ingram has served as President, CEO, and board member since 2017. He has no ambiguity about Sareptaâs mission: âWe know why we get up every day and work as hard as we do. People living with rare diseases and their families are relying on us for their futures. Weâre in a position to extend and change the lives of so many people. The opportunity to do good is just breathtaking.â DUCHENNE MUSCULAR DYSTROPHY (DMD) Duchenne muscular dystrophy, sometimes shortened to DMD or just Duchenne, is a rare genetic disease. It predominantly affects males, but, in rare cases, can also affect females. Duchenne causes the muscles in the body to become weak and damaged over time and iseventually fatal.
SAREPTA THERAPEUTICSâ INVESTIGATIONAL GENE THERAPY SRP -- Protein expression in muscle was sustained for two years following treatment in the low dose cohort, with mean beta-sarcoglycan expression of 54% at 24 months, compared to 36% at Day 60, as measuredby western blot --
GRANT AND SPONSORSHIP APPLICATION PORTAL Instructions for Sareptaâs Grant & Sponsorship Application Portal 5 of 32 8. After selecting an application type, you will be navigated to an Instructional Page, please read SAREPTA THERAPEUTICS TO SHARE CLINICAL UPDATE FOR SRP-5051 CAMBRIDGE, Mass., Dec. 04, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that on Monday, Dec. 7, 2020 at 8:30 am Eastern Time (ET), it will host a webcast and conference call to present interim data from the MOMENTUM study, a multiple-ascending dose clinical trial of SRP-5051 for the SRP-9004 (LGMD2D Î-SARCOGLYCAN) SRP-9004 (LGMD2D α-sarcoglycan) | Sarepta Corporate Rebuild. Main navigation. Our Disease Areas. Duchenne Muscular Dystrophy. Limb-girdle Muscular Dystrophy. Mucopolysaccharidosis Type IIIA. Charcot-Marie-Tooth Disease. Our Science. Gene Therapy Engine. 4045-301 CLINICAL TRIAL 4045-301 CLINICAL TRIAL For Duchenne muscular dystrophy (DMD) patients with deletion mu tations amenable to exon 45 or exon 53 skipping Information on site locations and SAREPTA THERAPEUTICS We see a revolution. Sarepta is a global biotechnology company on an urgent mission: engineer precision genetic medicine to reclaim futures otherwise impacted or cut short by rare diseases. Weâre ushering in a new era of drug development, with the goal of shortening the time from lab to patient, building the worldâs largest gene therapy ROUTE 79 SCHOLARSHIP PROGRAM Route 79, The Duchenne Scholarship Program, is designed to help students diagnosed with Duchenne muscular dystrophy pursue their post-high school educational goals. The scholarship is named after the79 exons in the dystrophin gene impacted by Duchenne. Sarepta knows that the route traveled by every person with Duchenne is distinct. SRP-9005 (LGMD2C Î-SARCOGLYCAN) SRP-9005 (LGMD2C Îł-sarcoglycan) | Sarepta Therapeutics. Main navigation. Our Disease Areas. Duchenne Muscular Dystrophy. Limb-girdle Muscular Dystrophy. Mucopolysaccharidosis Type IIIA. Charcot-Marie-Tooth Disease. Our Science. Gene Therapy Engine.INVESTOR RELATIONS
Investor Relations. At Sarepta, we are leading a revolution in precision genetic medicine and every day is an opportunity to change the lives of people living with rare disease. The Company has built an impressive position in Duchenne muscular dystrophy (DMD) and in gene therapies for limb-girdle muscular dystrophy diseases (LGMD), Charcot DUCHENNE MUSCULAR DYSTROPHY (DMD) Duchenne muscular dystrophy, sometimes shortened to DMD or just Duchenne, is a rare genetic disease. It predominantly affects males, but, in rare cases, can also affect females. Duchenne causes the muscles in the body to become weak and damaged over time and iseventually fatal.
SAREPTA THERAPEUTICSâ INVESTIGATIONAL GENE THERAPY SRP Sarepta Therapeuticsâ Investigational Gene Therapy SRP-9003 for the Treatment of Limb-Girdle Muscular Dystrophy Type 2E Shows Sustained Expression and Functional Improvements 2 Years After Administration LOUISE RODINO-KLAPAC, PHD Louise joined Sarepta in June 2018 and was appointed executive vice president, chief scientific officer in December 2020. Prior to this role Louise served as Sareptaâs senior vice president, gene therapy. She has led the design of most of Sareptaâs late-stage gene therapy candidates, has built and led Sareptaâs Gene Therapy Center of Excellence (GTCOE) in Columbus, Ohio, and has SAREPTA THERAPEUTICS TO SHARE CLINICAL UPDATE FOR SRP-5051SAREPTA THERAPEUTICS CAMBRIDGE MASAREPTA THERAPEUTICS INC LATEST NEWS CAMBRIDGE, Mass., Dec. 04, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that on Monday, Dec. 7, 2020 at 8:30 am Eastern Time (ET), it will host a webcast and conference call to present interim data from the MOMENTUM study, a multiple-ascending dose clinical trial of SRP-5051 for thePRE-CLINICAL
Emery-Dreifuss muscular dystrophy Type 1 (Columbia University) Read more about Emery-Dreifuss muscular dystrophy Type 1 (ColumbiaUniversity)
CLINICAL UPDATE: STUDY SRP-9001-103: 12-WEEK EXPRESSION « Back. Clinical Update: Study SRP-9001-103: 12-Week Expression and Safety Data Using Commercially Representative Material. 05/18/21 at8:30 AM EDT
SAREPTA THERAPEUTICS We see a revolution. Sarepta is a global biotechnology company on an urgent mission: engineer precision genetic medicine to reclaim futures otherwise impacted or cut short by rare diseases. Weâre ushering in a new era of drug development, with the goal of shortening the time from lab to patient, building the worldâs largest gene therapy ROUTE 79 SCHOLARSHIP PROGRAM Route 79, The Duchenne Scholarship Program, is designed to help students diagnosed with Duchenne muscular dystrophy pursue their post-high school educational goals. The scholarship is named after the79 exons in the dystrophin gene impacted by Duchenne. Sarepta knows that the route traveled by every person with Duchenne is distinct. SRP-9005 (LGMD2C Î-SARCOGLYCAN) SRP-9005 (LGMD2C Îł-sarcoglycan) | Sarepta Therapeutics. Main navigation. Our Disease Areas. Duchenne Muscular Dystrophy. Limb-girdle Muscular Dystrophy. Mucopolysaccharidosis Type IIIA. Charcot-Marie-Tooth Disease. Our Science. Gene Therapy Engine.INVESTOR RELATIONS
Investor Relations. At Sarepta, we are leading a revolution in precision genetic medicine and every day is an opportunity to change the lives of people living with rare disease. The Company has built an impressive position in Duchenne muscular dystrophy (DMD) and in gene therapies for limb-girdle muscular dystrophy diseases (LGMD), Charcot DUCHENNE MUSCULAR DYSTROPHY (DMD) Duchenne muscular dystrophy, sometimes shortened to DMD or just Duchenne, is a rare genetic disease. It predominantly affects males, but, in rare cases, can also affect females. Duchenne causes the muscles in the body to become weak and damaged over time and iseventually fatal.
SAREPTA THERAPEUTICSâ INVESTIGATIONAL GENE THERAPY SRP Sarepta Therapeuticsâ Investigational Gene Therapy SRP-9003 for the Treatment of Limb-Girdle Muscular Dystrophy Type 2E Shows Sustained Expression and Functional Improvements 2 Years After Administration LOUISE RODINO-KLAPAC, PHD Louise joined Sarepta in June 2018 and was appointed executive vice president, chief scientific officer in December 2020. Prior to this role Louise served as Sareptaâs senior vice president, gene therapy. She has led the design of most of Sareptaâs late-stage gene therapy candidates, has built and led Sareptaâs Gene Therapy Center of Excellence (GTCOE) in Columbus, Ohio, and has SAREPTA THERAPEUTICS TO SHARE CLINICAL UPDATE FOR SRP-5051SAREPTA THERAPEUTICS CAMBRIDGE MASAREPTA THERAPEUTICS INC LATEST NEWS CAMBRIDGE, Mass., Dec. 04, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that on Monday, Dec. 7, 2020 at 8:30 am Eastern Time (ET), it will host a webcast and conference call to present interim data from the MOMENTUM study, a multiple-ascending dose clinical trial of SRP-5051 for thePRE-CLINICAL
Emery-Dreifuss muscular dystrophy Type 1 (Columbia University) Read more about Emery-Dreifuss muscular dystrophy Type 1 (ColumbiaUniversity)
CLINICAL UPDATE: STUDY SRP-9001-103: 12-WEEK EXPRESSION « Back. Clinical Update: Study SRP-9001-103: 12-Week Expression and Safety Data Using Commercially Representative Material. 05/18/21 at8:30 AM EDT
COVID-19 RESOURCES FOR PATIENTS AND FAMILIES Sarepta continues to provide an uninterrupted supply of its therapies to patients and is working to do everything reasonably possible to minimize disruption to patients and meet their needs during this uncertain time. This page is intended as a resource to gather relevant links and information in one place. It is critically important for patients and caregivers to contact your physician withPIPELINE & PRODUCTS
Pipeline & Products | Sarepta Therapeutics. At Sarepta, we are working with urgency to develop breakthrough therapies to treat genetic diseases. Currently, we have more than 40 investigational therapies in various stages of developmentâmany already in late-stage clinical trials. In many cases, development is being accelerated by our gene NEWS & PRESS RELEASES Read news and articles from Sarepta, a global biotechnology company developing potentially life-changing precision genetic medicine.OUR PIPELINE
See our pipeline for RNA technologies, gene therapy, and gene editing to learn about our research in rare diseases.MEET OUR LEADERSHIP
Gilmore O'Neill, MB, MMSC. Executive Vice President, R&D and Chief Medical Officer. âI met the parents of a young boy with Duchenne muscular dystrophy. They said, âWeâre so grateful youâre developing this gene therapy, but weâre really worried because our son is deteriorating fast.â. Well, we have a sort of sacredresponsibility to
SAREPTASSIST
SareptAssist | Sarepta Corporate Rebuild. Support, By Your Side. SareptAssist is a patient support program designed to provide you with information to help you navigate the process of starting and staying on therapy. Our dedicated team will provide information on: Insurance Benefits. Financial Assistance Options. Treatment Logistics. SAREPTA THERAPEUTICSâ INVESTIGATIONAL GENE THERAPY SRP -- Protein expression in muscle was sustained for two years following treatment in the low dose cohort, with mean beta-sarcoglycan expression of 54% at 24 months, compared to 36% at Day 60, as measuredby western blot --
SAREPTA THERAPEUTICS TO PRESENT AT UPCOMING INVESTOR CAMBRIDGE, Mass., June 03, 2021 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that senior management will participate in fireside chats at two upcoming virtual investorconferences:
SAREPTA THERAPEUTICS TO SHARE CLINICAL UPDATE FOR SRP-5051 CAMBRIDGE, Mass., Dec. 04, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that on Monday, Dec. 7, 2020 at 8:30 am Eastern Time (ET), it will host a webcast and conference call to present interim data from the MOMENTUM study, a multiple-ascending dose clinical trial of SRP-5051 for the SAREPTA THERAPEUTICS ANNOUNCES PARTNERSHIP WITH ROCHE IN â Roche obtains the exclusive right to launch and commercialize SRP-9001 outside the United States â â At closing, Sarepta will receive an upfront payment of $1.15 billion, comprising $750 million in cash and $400 million in Sarepta stock, priced at $158.59 per share of common stock â â Additionally, Sarepta is eligible to receive up to $1.7 billion in regulatory and sales milestones SAREPTA THERAPEUTICS We see a revolution. Sarepta is a global biotechnology company on an urgent mission: engineer precision genetic medicine to reclaim futures otherwise impacted or cut short by rare diseases. Weâre ushering in a new era of drug development, with the goal of shortening the time from lab to patient, building the worldâs largest gene therapy ROUTE 79 SCHOLARSHIP PROGRAM Route 79, The Duchenne Scholarship Program, is designed to help students diagnosed with Duchenne muscular dystrophy pursue their post-high school educational goals. The scholarship is named after the79 exons in the dystrophin gene impacted by Duchenne. Sarepta knows that the route traveled by every person with Duchenne is distinct. SRP-9005 (LGMD2C Î-SARCOGLYCAN) SRP-9005 (LGMD2C Îł-sarcoglycan) | Sarepta Therapeutics. Main navigation. Our Disease Areas. Duchenne Muscular Dystrophy. Limb-girdle Muscular Dystrophy. Mucopolysaccharidosis Type IIIA. Charcot-Marie-Tooth Disease. Our Science. Gene Therapy Engine.INVESTOR RELATIONS
Investor Relations. At Sarepta, we are leading a revolution in precision genetic medicine and every day is an opportunity to change the lives of people living with rare disease. The Company has built an impressive position in Duchenne muscular dystrophy (DMD) and in gene therapies for limb-girdle muscular dystrophy diseases (LGMD), Charcot DUCHENNE MUSCULAR DYSTROPHY (DMD) Duchenne muscular dystrophy, sometimes shortened to DMD or just Duchenne, is a rare genetic disease. It predominantly affects males, but, in rare cases, can also affect females. Duchenne causes the muscles in the body to become weak and damaged over time and iseventually fatal.
SAREPTA THERAPEUTICSâ INVESTIGATIONAL GENE THERAPY SRP Sarepta Therapeuticsâ Investigational Gene Therapy SRP-9003 for the Treatment of Limb-Girdle Muscular Dystrophy Type 2E Shows Sustained Expression and Functional Improvements 2 Years After Administration LOUISE RODINO-KLAPAC, PHD Louise joined Sarepta in June 2018 and was appointed executive vice president, chief scientific officer in December 2020. Prior to this role Louise served as Sareptaâs senior vice president, gene therapy. She has led the design of most of Sareptaâs late-stage gene therapy candidates, has built and led Sareptaâs Gene Therapy Center of Excellence (GTCOE) in Columbus, Ohio, and has SAREPTA THERAPEUTICS TO SHARE CLINICAL UPDATE FOR SRP-5051SAREPTA THERAPEUTICS CAMBRIDGE MASAREPTA THERAPEUTICS INC LATEST NEWS CAMBRIDGE, Mass., Dec. 04, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that on Monday, Dec. 7, 2020 at 8:30 am Eastern Time (ET), it will host a webcast and conference call to present interim data from the MOMENTUM study, a multiple-ascending dose clinical trial of SRP-5051 for thePRE-CLINICAL
Emery-Dreifuss muscular dystrophy Type 1 (Columbia University) Read more about Emery-Dreifuss muscular dystrophy Type 1 (ColumbiaUniversity)
CLINICAL UPDATE: STUDY SRP-9001-103: 12-WEEK EXPRESSION « Back. Clinical Update: Study SRP-9001-103: 12-Week Expression and Safety Data Using Commercially Representative Material. 05/18/21 at8:30 AM EDT
SAREPTA THERAPEUTICS We see a revolution. Sarepta is a global biotechnology company on an urgent mission: engineer precision genetic medicine to reclaim futures otherwise impacted or cut short by rare diseases. Weâre ushering in a new era of drug development, with the goal of shortening the time from lab to patient, building the worldâs largest gene therapy ROUTE 79 SCHOLARSHIP PROGRAM Route 79, The Duchenne Scholarship Program, is designed to help students diagnosed with Duchenne muscular dystrophy pursue their post-high school educational goals. The scholarship is named after the79 exons in the dystrophin gene impacted by Duchenne. Sarepta knows that the route traveled by every person with Duchenne is distinct. SRP-9005 (LGMD2C Î-SARCOGLYCAN) SRP-9005 (LGMD2C Îł-sarcoglycan) | Sarepta Therapeutics. Main navigation. Our Disease Areas. Duchenne Muscular Dystrophy. Limb-girdle Muscular Dystrophy. Mucopolysaccharidosis Type IIIA. Charcot-Marie-Tooth Disease. Our Science. Gene Therapy Engine.INVESTOR RELATIONS
Investor Relations. At Sarepta, we are leading a revolution in precision genetic medicine and every day is an opportunity to change the lives of people living with rare disease. The Company has built an impressive position in Duchenne muscular dystrophy (DMD) and in gene therapies for limb-girdle muscular dystrophy diseases (LGMD), Charcot DUCHENNE MUSCULAR DYSTROPHY (DMD) Duchenne muscular dystrophy, sometimes shortened to DMD or just Duchenne, is a rare genetic disease. It predominantly affects males, but, in rare cases, can also affect females. Duchenne causes the muscles in the body to become weak and damaged over time and iseventually fatal.
SAREPTA THERAPEUTICSâ INVESTIGATIONAL GENE THERAPY SRP Sarepta Therapeuticsâ Investigational Gene Therapy SRP-9003 for the Treatment of Limb-Girdle Muscular Dystrophy Type 2E Shows Sustained Expression and Functional Improvements 2 Years After Administration LOUISE RODINO-KLAPAC, PHD Louise joined Sarepta in June 2018 and was appointed executive vice president, chief scientific officer in December 2020. Prior to this role Louise served as Sareptaâs senior vice president, gene therapy. She has led the design of most of Sareptaâs late-stage gene therapy candidates, has built and led Sareptaâs Gene Therapy Center of Excellence (GTCOE) in Columbus, Ohio, and has SAREPTA THERAPEUTICS TO SHARE CLINICAL UPDATE FOR SRP-5051SAREPTA THERAPEUTICS CAMBRIDGE MASAREPTA THERAPEUTICS INC LATEST NEWS CAMBRIDGE, Mass., Dec. 04, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that on Monday, Dec. 7, 2020 at 8:30 am Eastern Time (ET), it will host a webcast and conference call to present interim data from the MOMENTUM study, a multiple-ascending dose clinical trial of SRP-5051 for thePRE-CLINICAL
Emery-Dreifuss muscular dystrophy Type 1 (Columbia University) Read more about Emery-Dreifuss muscular dystrophy Type 1 (ColumbiaUniversity)
CLINICAL UPDATE: STUDY SRP-9001-103: 12-WEEK EXPRESSION « Back. Clinical Update: Study SRP-9001-103: 12-Week Expression and Safety Data Using Commercially Representative Material. 05/18/21 at8:30 AM EDT
COVID-19 RESOURCES FOR PATIENTS AND FAMILIES Sarepta continues to provide an uninterrupted supply of its therapies to patients and is working to do everything reasonably possible to minimize disruption to patients and meet their needs during this uncertain time. This page is intended as a resource to gather relevant links and information in one place. It is critically important for patients and caregivers to contact your physician withPIPELINE & PRODUCTS
Pipeline & Products | Sarepta Therapeutics. At Sarepta, we are working with urgency to develop breakthrough therapies to treat genetic diseases. Currently, we have more than 40 investigational therapies in various stages of developmentâmany already in late-stage clinical trials. In many cases, development is being accelerated by our gene NEWS & PRESS RELEASES Read news and articles from Sarepta, a global biotechnology company developing potentially life-changing precision genetic medicine.OUR PIPELINE
See our pipeline for RNA technologies, gene therapy, and gene editing to learn about our research in rare diseases.MEET OUR LEADERSHIP
Gilmore O'Neill, MB, MMSC. Executive Vice President, R&D and Chief Medical Officer. âI met the parents of a young boy with Duchenne muscular dystrophy. They said, âWeâre so grateful youâre developing this gene therapy, but weâre really worried because our son is deteriorating fast.â. Well, we have a sort of sacredresponsibility to
SAREPTASSIST
SareptAssist | Sarepta Corporate Rebuild. Support, By Your Side. SareptAssist is a patient support program designed to provide you with information to help you navigate the process of starting and staying on therapy. Our dedicated team will provide information on: Insurance Benefits. Financial Assistance Options. Treatment Logistics. SAREPTA THERAPEUTICSâ INVESTIGATIONAL GENE THERAPY SRP -- Protein expression in muscle was sustained for two years following treatment in the low dose cohort, with mean beta-sarcoglycan expression of 54% at 24 months, compared to 36% at Day 60, as measuredby western blot --
SAREPTA THERAPEUTICS TO PRESENT AT UPCOMING INVESTOR CAMBRIDGE, Mass., June 03, 2021 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that senior management will participate in fireside chats at two upcoming virtual investorconferences:
SAREPTA THERAPEUTICS TO SHARE CLINICAL UPDATE FOR SRP-5051 CAMBRIDGE, Mass., Dec. 04, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that on Monday, Dec. 7, 2020 at 8:30 am Eastern Time (ET), it will host a webcast and conference call to present interim data from the MOMENTUM study, a multiple-ascending dose clinical trial of SRP-5051 for the SAREPTA THERAPEUTICS ANNOUNCES PARTNERSHIP WITH ROCHE IN â Roche obtains the exclusive right to launch and commercialize SRP-9001 outside the United States â â At closing, Sarepta will receive an upfront payment of $1.15 billion, comprising $750 million in cash and $400 million in Sarepta stock, priced at $158.59 per share of common stock â â Additionally, Sarepta is eligible to receive up to $1.7 billion in regulatory and sales milestones SAREPTA THERAPEUTICS We see a revolution. Sarepta is a global biotechnology company on an urgent mission: engineer precision genetic medicine to reclaim futures otherwise impacted or cut short by rare diseases. Weâre ushering in a new era of drug development, with the goal of shortening the time from lab to patient, building the worldâs largest gene therapy ROUTE 79 SCHOLARSHIP PROGRAM Route 79, The Duchenne Scholarship Program, is designed to help students diagnosed with Duchenne muscular dystrophy pursue their post-high school educational goals. The scholarship is named after the79 exons in the dystrophin gene impacted by Duchenne. Sarepta knows that the route traveled by every person with Duchenne is distinct. SRP-9005 (LGMD2C Î-SARCOGLYCAN) SRP-9005 (LGMD2C Îł-sarcoglycan) | Sarepta Therapeutics. Main navigation. Our Disease Areas. Duchenne Muscular Dystrophy. Limb-girdle Muscular Dystrophy. Mucopolysaccharidosis Type IIIA. Charcot-Marie-Tooth Disease. Our Science. Gene Therapy Engine.INVESTOR RELATIONS
Investor Relations. At Sarepta, we are leading a revolution in precision genetic medicine and every day is an opportunity to change the lives of people living with rare disease. The Company has built an impressive position in Duchenne muscular dystrophy (DMD) and in gene therapies for limb-girdle muscular dystrophy diseases (LGMD), Charcot DUCHENNE MUSCULAR DYSTROPHY (DMD) Duchenne muscular dystrophy, sometimes shortened to DMD or just Duchenne, is a rare genetic disease. It predominantly affects males, but, in rare cases, can also affect females. Duchenne causes the muscles in the body to become weak and damaged over time and iseventually fatal.
SAREPTA THERAPEUTICSâ INVESTIGATIONAL GENE THERAPY SRP Sarepta Therapeuticsâ Investigational Gene Therapy SRP-9003 for the Treatment of Limb-Girdle Muscular Dystrophy Type 2E Shows Sustained Expression and Functional Improvements 2 Years After Administration LOUISE RODINO-KLAPAC, PHD Louise joined Sarepta in June 2018 and was appointed executive vice president, chief scientific officer in December 2020. Prior to this role Louise served as Sareptaâs senior vice president, gene therapy. She has led the design of most of Sareptaâs late-stage gene therapy candidates, has built and led Sareptaâs Gene Therapy Center of Excellence (GTCOE) in Columbus, Ohio, and has SAREPTA THERAPEUTICS TO SHARE CLINICAL UPDATE FOR SRP-5051SAREPTA THERAPEUTICS CAMBRIDGE MASAREPTA THERAPEUTICS INC LATEST NEWS CAMBRIDGE, Mass., Dec. 04, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that on Monday, Dec. 7, 2020 at 8:30 am Eastern Time (ET), it will host a webcast and conference call to present interim data from the MOMENTUM study, a multiple-ascending dose clinical trial of SRP-5051 for thePRE-CLINICAL
Emery-Dreifuss muscular dystrophy Type 1 (Columbia University) Read more about Emery-Dreifuss muscular dystrophy Type 1 (ColumbiaUniversity)
CLINICAL UPDATE: STUDY SRP-9001-103: 12-WEEK EXPRESSION « Back. Clinical Update: Study SRP-9001-103: 12-Week Expression and Safety Data Using Commercially Representative Material. 05/18/21 at8:30 AM EDT
SAREPTA THERAPEUTICS We see a revolution. Sarepta is a global biotechnology company on an urgent mission: engineer precision genetic medicine to reclaim futures otherwise impacted or cut short by rare diseases. Weâre ushering in a new era of drug development, with the goal of shortening the time from lab to patient, building the worldâs largest gene therapy ROUTE 79 SCHOLARSHIP PROGRAM Route 79, The Duchenne Scholarship Program, is designed to help students diagnosed with Duchenne muscular dystrophy pursue their post-high school educational goals. The scholarship is named after the79 exons in the dystrophin gene impacted by Duchenne. Sarepta knows that the route traveled by every person with Duchenne is distinct. SRP-9005 (LGMD2C Î-SARCOGLYCAN) SRP-9005 (LGMD2C Îł-sarcoglycan) | Sarepta Therapeutics. Main navigation. Our Disease Areas. Duchenne Muscular Dystrophy. Limb-girdle Muscular Dystrophy. Mucopolysaccharidosis Type IIIA. Charcot-Marie-Tooth Disease. Our Science. Gene Therapy Engine.INVESTOR RELATIONS
Investor Relations. At Sarepta, we are leading a revolution in precision genetic medicine and every day is an opportunity to change the lives of people living with rare disease. The Company has built an impressive position in Duchenne muscular dystrophy (DMD) and in gene therapies for limb-girdle muscular dystrophy diseases (LGMD), Charcot DUCHENNE MUSCULAR DYSTROPHY (DMD) Duchenne muscular dystrophy, sometimes shortened to DMD or just Duchenne, is a rare genetic disease. It predominantly affects males, but, in rare cases, can also affect females. Duchenne causes the muscles in the body to become weak and damaged over time and iseventually fatal.
SAREPTA THERAPEUTICSâ INVESTIGATIONAL GENE THERAPY SRP Sarepta Therapeuticsâ Investigational Gene Therapy SRP-9003 for the Treatment of Limb-Girdle Muscular Dystrophy Type 2E Shows Sustained Expression and Functional Improvements 2 Years After Administration LOUISE RODINO-KLAPAC, PHD Louise joined Sarepta in June 2018 and was appointed executive vice president, chief scientific officer in December 2020. Prior to this role Louise served as Sareptaâs senior vice president, gene therapy. She has led the design of most of Sareptaâs late-stage gene therapy candidates, has built and led Sareptaâs Gene Therapy Center of Excellence (GTCOE) in Columbus, Ohio, and has SAREPTA THERAPEUTICS TO SHARE CLINICAL UPDATE FOR SRP-5051SAREPTA THERAPEUTICS CAMBRIDGE MASAREPTA THERAPEUTICS INC LATEST NEWS CAMBRIDGE, Mass., Dec. 04, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that on Monday, Dec. 7, 2020 at 8:30 am Eastern Time (ET), it will host a webcast and conference call to present interim data from the MOMENTUM study, a multiple-ascending dose clinical trial of SRP-5051 for thePRE-CLINICAL
Emery-Dreifuss muscular dystrophy Type 1 (Columbia University) Read more about Emery-Dreifuss muscular dystrophy Type 1 (ColumbiaUniversity)
CLINICAL UPDATE: STUDY SRP-9001-103: 12-WEEK EXPRESSION « Back. Clinical Update: Study SRP-9001-103: 12-Week Expression and Safety Data Using Commercially Representative Material. 05/18/21 at8:30 AM EDT
COVID-19 RESOURCES FOR PATIENTS AND FAMILIES Sarepta continues to provide an uninterrupted supply of its therapies to patients and is working to do everything reasonably possible to minimize disruption to patients and meet their needs during this uncertain time. This page is intended as a resource to gather relevant links and information in one place. It is critically important for patients and caregivers to contact your physician withPIPELINE & PRODUCTS
Pipeline & Products | Sarepta Therapeutics. At Sarepta, we are working with urgency to develop breakthrough therapies to treat genetic diseases. Currently, we have more than 40 investigational therapies in various stages of developmentâmany already in late-stage clinical trials. In many cases, development is being accelerated by our gene NEWS & PRESS RELEASES Read news and articles from Sarepta, a global biotechnology company developing potentially life-changing precision genetic medicine.OUR PIPELINE
See our pipeline for RNA technologies, gene therapy, and gene editing to learn about our research in rare diseases.MEET OUR LEADERSHIP
Gilmore O'Neill, MB, MMSC. Executive Vice President, R&D and Chief Medical Officer. âI met the parents of a young boy with Duchenne muscular dystrophy. They said, âWeâre so grateful youâre developing this gene therapy, but weâre really worried because our son is deteriorating fast.â. Well, we have a sort of sacredresponsibility to
SAREPTASSIST
SareptAssist | Sarepta Corporate Rebuild. Support, By Your Side. SareptAssist is a patient support program designed to provide you with information to help you navigate the process of starting and staying on therapy. Our dedicated team will provide information on: Insurance Benefits. Financial Assistance Options. Treatment Logistics. SAREPTA THERAPEUTICSâ INVESTIGATIONAL GENE THERAPY SRP -- Protein expression in muscle was sustained for two years following treatment in the low dose cohort, with mean beta-sarcoglycan expression of 54% at 24 months, compared to 36% at Day 60, as measuredby western blot --
SAREPTA THERAPEUTICS TO PRESENT AT UPCOMING INVESTOR CAMBRIDGE, Mass., June 03, 2021 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that senior management will participate in fireside chats at two upcoming virtual investorconferences:
SAREPTA THERAPEUTICS TO SHARE CLINICAL UPDATE FOR SRP-5051 CAMBRIDGE, Mass., Dec. 04, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that on Monday, Dec. 7, 2020 at 8:30 am Eastern Time (ET), it will host a webcast and conference call to present interim data from the MOMENTUM study, a multiple-ascending dose clinical trial of SRP-5051 for the SAREPTA THERAPEUTICS ANNOUNCES PARTNERSHIP WITH ROCHE IN â Roche obtains the exclusive right to launch and commercialize SRP-9001 outside the United States â â At closing, Sarepta will receive an upfront payment of $1.15 billion, comprising $750 million in cash and $400 million in Sarepta stock, priced at $158.59 per share of common stock â â Additionally, Sarepta is eligible to receive up to $1.7 billion in regulatory and sales milestones Skip to main contentPRESS RELEASES
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05/18/2021 SAREPTA THERAPEUTICSâ INVESTIGATIONAL GENE THERAPY FOR THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY, SRP-9001, DEMONSTRATES ROBUST EXPRESSION AND CONSISTENT SAFETY PROFILE USING SAREPTAâS COMMERCIAL PROCESS MATERIALUTILITY MENU
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CELEBRATING
OUR
PRIDE
At Sarepta, we strive to promote diversity, inclusion, equal opportunity and personal development across the organization. Our Employee Resource Group Pride created by employees who identify as LGBTQIA+ along with their allies provides members with a forum to promote a dynamic, welcoming, and friendly atmosphere while strengthening diversity and inclusion at Sarepta.*
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SOME SEE SLOW AND STEADY SCIENTIFIC PROGRESS. WE SEE AÂ REVOLUTION. Sarepta is a global biotechnology company on an urgent mission: engineer precision genetic medicine to reclaim futures otherwise impacted or cut short by rare diseases. Weâre ushering in a new era of drug development, with the goal of shortening the time from lab to patient, building the worldâs largest gene therapy manufacturing capacity, and rethinking access and reimbursement models for revolutionary new treatments. We are in a daily race to transform genetic understanding into genetic medicine. Because every day is an opportunity to change the lives of people living with rare disease.Learn more
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SOME THINK A CURE FOR A RARE DISEASE IS A MIRACLE. OUR SCIENTISTS WOULD DISAGREE. Sarepta is engineering solutions for rare diseases with science that is on the forefront of precision genetic medicine. Our platforms include: gene therapy, RNA technologies, and gene editing. We are in a daily race to save lives stolen or impacted by rare disease.Learn more
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PATIENTS CANâT WAIT FOR THE NEXT BREAKTHROUGH IN MEDICAL RESEARCH.SO NEITHER WILL WE.
While there may be as many as 7,000 rare diseases, only a small percentage have treatments. That is why we are relentless in our dedication: Leverage the best science to help as many patients as possible. Today, we are doing just that in Duchenne muscular dystrophy, in six forms of limb-girdle muscular dystrophy, in Charcot-Marie-Tooth disease, and in Mucopolysaccharidosis type IIIA (MPS IIIA), among others. Sarepta will always follow the science and continuously evaluate other diseases and modalities to pursue.Learn more
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THE OPPORTUNITY TO TRANSFORMÂ LIVES IS BREATHTAKING.JOIN US.
We see endless possibilities for changing the future of medicine. Weâre looking for people who see unlimited potential in themselves, opportunity where others see roadblocks, and solutions limited only by the power of imagination and the drive to do things differently. Learn more about working at Sarepta* 1
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RESOURCES FOR PATIENTS & CAREGIVERS IN RESPONSE TO COVID-19Learn More
CELEBRATING OUR SAREPTA TEAM AND EXTENDED FAMILY Behind the exceptional group of individuals who make up our Sarepta team is a support system of partners, parents, siblings, spouses, children and roommates. This groupâs incredible support has been is more apparent and meaningful now than ever. Several teammates let us into their homes virtually to share what work and life has been like during the pandemic.PRESS RELEASES
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06/03/2021 SAREPTA THERAPEUTICS TO PRESENT AT UPCOMING INVESTORCONFERENCES
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05/28/2021 SAREPTA THERAPEUTICS ANNOUNCES INDUCEMENT GRANTS UNDER NASDAQ LISTING RULE 5635(C)(4)*
05/18/2021 SAREPTA THERAPEUTICSâ INVESTIGATIONAL GENE THERAPY FOR THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY, SRP-9001, DEMONSTRATES ROBUST EXPRESSION AND CONSISTENT SAFETY PROFILE USING SAREPTAâS COMMERCIAL PROCESS MATERIAL SPOTLIGHT ON: DEVELOPMENT PROGRAMS EXPLORING THE POTENTIAL OF GENE THERAPIES FOR LIMB-GIRDLE MUSCULARDYSTROPHIES
Dr. Louise Rodino-Klapac, discusses the science of limb-girdle muscular dystrophies (LGMDs) and how our gene therapy engine will help engineer potential therapies for this group of inherited neuromuscular diseases. RNA-TARGETED THERAPIES: A DEVELOPMENT STRATEGY FOR PROTEINUPREGULATION
Learn more about the biochemical structure of phosphodiamidate morpholino oligomers (PMOs) and PPMOs and their mechanism.CORPORATE UPDATES
A NEW FDA-APPROVED TREATMENT View our approved productsRead press release
SAREPTA THERAPEUTICS' PRESENTATION FROM THE 39TH ANNUAL J.P. MORGAN HEALTHCARE CONFERENCE (2021) View presentation (PDF)*
Sarepta Therapeutics @SareptaTwitter Logo As we listen to the stories of those living with #LimbGirdle#MuscularDystrophy
, one thing is
clear: experiences and symptom onset can vastly differ for each individual, making awareness even more important. Read the stories of those living with #LGMD : limbgirdle.com/lgmd-stories* Reply
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4 Jun 2021
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Sarepta Therapeutics @SareptaTwitter Logo We're proud to support @MDAorg 's#LimbGirdle
#MuscularDystrophy
Symposium and hope you can join virtually on Saturday, June 5. #LGMDMDA USA
@MDAorg
THIS SATURDAY, join our #MDA Engage #LGMD #MuscularDystrophy Symposium, June 5, 11-4:30pm ET. Topics: #ClinicalTrials, cardiology, exercise, and more w/ experts @pbkang7, @MKIttlesonMD, @CedarsSinai, @umnmedschool, @seattlechildren, @uihealthcare https://t.co/SNfDGSSaCF https://t.co/KGQpK6Lcbn* Reply
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4 Jun 2021
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Sarepta Therapeutics @SareptaTwitter Logo After learning he has LGMD, Greg felt depressed and scared of the future. Read how he has learned to channel those feelings to be able to live in a way that allows him to be grateful for the simple things:bit.ly/3fLUjE0
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3 Jun 2021
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