Rare disease drugmaker · based in Cambridge, United States · founded 1980 · ticker SRPT · sarepta.com
Sarepta Therapeutics works on 5 rare diseases we track, including Duchenne Muscular Dystrophy, Facioscapulohumeral Muscular Dystrophy and 3 more. It has 9 active clinical trials, and 3 are looking for volunteers now. It also has 4 FDA-approved rare disease drugs.
The FDA is due to decide on Amondys 45 + Vyondys 53 for Duchenne muscular dystrophy (conversion to traditional approval) by February 28, 2027.
Sarepta Therapeutics is a Cambridge, Massachusetts biotech that has spent most of the last decade trying to deliver gene therapy to patients with Duchenne muscular dystrophy. The company was originally founded in 1980 and trades on the NASDAQ under the ticker SRPT.
Sarepta Therapeutics pipeline
Each row lists the diseases Sarepta Therapeutics is studying at that stage, from the ones closest to approval down to the earliest work.
These are Sarepta Therapeutics trials for the rare diseases we track, from ClinicalTrials.gov. Statuses there can lag behind company news, so confirm with the study team before you plan around one.
3 trials looking for volunteers now
Looking for volunteersPhase 4Recently updatedNCT07542314
Study to Evaluate the Safety and Effectiveness of ELEVIDYS in Participants With Duchenne Muscular Dystrophy Treated in a Post-Marketing Setting
The primary objective of this study is to evaluate acute liver injury (ALI) rates associated with ELEVIDYS with the addition of sirolimus as an adjunct prophylactic immunosuppression agent.
Looking for volunteersPhase 1Recently updatedNCT04626674
A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) - Non-Ambulatory Cohort
Cohort 8 (non-ambulatory participants) is currently enrolling new participants. Enrollment for Cohorts 1 through 7 has been completed.
This is an open-label gene transfer therapy study evaluating the safety of and expression from delandistrogene moxeparvovec in participants with Duchenne Muscular Dystrophy (DMD). The maximum participant duration for this study is 156 weeks.
Underway, not taking new volunteersPhase 2Recently updatedNCT06128564
A Gene Delivery Study to Evaluate the Safety and Expression of Delandistrogene Moxeparvovec in Participants Under the Age of Four With Duchenne Muscular Dystrophy (DMD)
This open-label, single-arm study will evaluate the safety and expression of delandistrogene moxeparvovec in participants with DMD. Participants will be in the study for approximately 264 weeks.
Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approach to identify affected infants, 2) address the impact on infants and families who screen positive, and 3) evaluate the Early Check program. The Early Check screening will lead to earlier identification of newborns with rare health conditions in addition to providing important data on the implementation of this model program. Early diagnosis may result in health and development benefits for the newborns. Infants who have newborn screening in North Carolina will be eligible to participate, equating to over 120,000 eligible infants a year. Over 95% of participants are expected to screen negative. Newborns who screen positive and their parents are invited to additional research activities and services. Parents can enroll eligible newborns on the Early Check electronic Research Portal. Screening tests are conducted on residual blood from existing newborn screening dried blood spots. Confirmatory testing is provided free-of-charge for infants who screen positive, and carrier testing is provided to mothers of infants with fragile X. Affected newborns have a physical and developmental evaluation. Their parents have genetic counseling and are invited to participate in surveys and interviews. Ongoing evaluation of the program includes additional parent interviews.
Underway, not taking new volunteersPhase 3Updated a few months agoNCT05881408
A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD)
The study will evaluate the safety and efficacy of delandistrogene moxeparvovec gene transfer therapy in non-ambulatory and ambulatory males with DMD. This is a randomized, double-blind, placebo-controlled 2-part study. Participants will be in the study for approximately 128 weeks. All participants will have the opportunity to receive intravenous (IV) delandistrogene moxeparvovec in either Part 1 or Part 2.
Underway, not taking new volunteersPhase 3No updates in a whileNCT06246513
A Trial to Learn More About an Experimental Gene Therapy Called Bidridistrogene Xeboparvovec (SRP-9003) as a Possible Treatment for Limb Girdle Muscular Dystrophy 2E/R4
This is a multicenter, global study of the effects of a single systemic dose of SRP-9003 on beta-sarcoglycan (β-SG) gene expression in participants with limb-girdle muscular dystrophy, type 2E/R4 (LGMD2E/R4). This study will consist of both ambulatory participants (Cohort 1) and non-ambulatory participants (Cohort 2).
Underway, not taking new volunteersPhase 1No updates in a whileNCT05876780
A Gene Transfer Single Dose Study to Evaluate the Safety, Tolerability and Efficacy of SRP-9003 in Non-Ambulatory and Ambulatory Participants With Limb Girdle Muscular Dystrophy, Type 2E/R4 (Beta-Sarcoglycan [β-SG] Deficiency)
The primary purpose of this study is to evaluate the safety of SRP-9003 and to quantify expression of β-SG in the skeletal muscle of participants with limb-girdle muscular dystrophy, type 2E/R4 (LGMD2E/R4). The study will include both ambulatory (Cohort 1) and non-ambulatory (Cohort 2) participants.
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Today its identity is built around the modern Sarepta of the 2010s onward, a company that has bet repeatedly that genetic medicine for neuromuscular diseases is worth pursuing even when the path through the FDA is hard.
The flagship product is Elevidys (delandistrogene moxeparvovec), the first gene therapy approved for Duchenne muscular dystrophy. Duchenne is a rare, progressive genetic disease that primarily affects boys, caused by mutations on the X chromosome. Patients are missing functional dystrophin, a protein their muscles need to repair themselves, so muscle tissue is gradually replaced by scar and fat over time.
Most patients lose the ability to walk before adolescence and face premature death from cardiac and respiratory complications. Elevidys delivers a shortened working version of the dystrophin gene to muscle cells using a viral vector called AAV. The drug received FDA accelerated approval in 2023 for ambulatory boys aged 4 to 5, and the indication was expanded in 2024.
The Elevidys story has had a difficult more recent arc. In 2025, two non-ambulatory pediatric patients died from acute liver failure following Elevidys treatment. In November 2025 the FDA approved a Boxed Warning on the drug's label and revised the approved indication to limit use to ambulatory patients aged 4 and older.
Keep reading about Sarepta Therapeutics
The FDA also required Sarepta to run an observational study of about 200 patients that checks liver function for at least 12 months after the infusion. For boys who can no longer walk, Sarepta is testing an added immune-suppressing medicine called sirolimus in Cohort 8 of its ENDEAVOR study, with full enrollment expected by the end of 2026.
The events have made Elevidys a difficult and personal decision for many families weighing risk against the natural course of the disease, and Sarepta has been in active dialogue with the FDA and the DMD patient community since.
Sarepta also markets three earlier antisense oligonucleotide therapies for DMD that work by skipping over specific mutated exons of the dystrophin gene so a partially functional protein can still be produced. These are Exondys 51, Vyondys 53, and Amondys 45, each designed for a different DMDmutation type. In July 2025 Sarepta paused most of its limb-girdle muscular dystrophy gene therapies to cut costs, keeping SRP-9003 for LGMD type 2E/R4.
That same month the FDA placed Sarepta's LGMD gene therapy trials, SRP-9003 among them, on clinical hold after a patient in one of its LGMD gene therapy trials died of acute liver failure. BioSpace reported on October 15, 2025 that the SRP-9003 hold was still in place, and Sarepta has not announced since then that it was lifted.
Its pipeline now centers on siRNA medicines for facioscapulohumeral dystrophy, myotonic dystrophy type 1 and Huntington disease, with dosing underway in a first-in-human Huntington study.
Where Sarepta Therapeutics trials take place
The trials above run at 111 study sites in 19 countries. Tap a country to see its states, cities and hospitals.
United States
46▼
United Kingdom
9▼
Italy
9▼
Belgium
7▼
Germany
7▼
Spain
5▼
Australia
4▼
Canada
4▼
South Korea
4▼
Japan
3▼
Sweden
3▼
Israel
2▼
Rare diseases Sarepta Therapeutics works on
Each disease page lists every open trial, not only Sarepta Therapeutics's, plus approved treatments.
Facioscapulohumeral Muscular Dystrophy (FSHD) is an inherited muscular dystrophy characterized by progressive weakness of the facial, shoulder, and upper arm muscles.
How common: About 1 in 20,000 people (MedlinePlus Genetics); one of the most common hereditary muscular dystrophies
Huntington disease is an autosomal dominantneurodegenerative disorder caused by an expanded CAG trinucleotide repeat in the huntingtin gene.
How common: About 3-7 per 100,000 people of European ancestry, according to MedlinePlus Genetics; less common in people of Japanese, Chinese and African descent
Limb-Girdle Muscular Dystrophy (LGMD) refers to a genetically heterogeneous group of muscular dystrophies characterized by progressive weakness of the hip and shoulder girdle muscles.
How common: Approximately 1 per 14,500 to 1 per 123,000 people depending on geographic region; exact prevalence varies by subtype
Myotonic Dystrophy (DM) is the most common muscular dystrophy in adults, characterized by progressive muscle weakness and myotonia—the inability of muscles to relax after contraction.
How common: Approximately 1 in 3,000 to 1 in 8,000 people globally; higher prevalence in some populations
Where to get support
Patient groups can explain a diagnosis, point you to specialists and tell you about trials before they open. Sarepta Therapeutics's own site is sarepta.com, and our help paying for treatment page lists drugmaker programs and charity funds.
1 shared disease, including Duchenne Muscular Dystrophy
Questions about Sarepta Therapeutics
Sarepta Therapeutics is a Cambridge, Massachusetts biotech that has spent most of the last decade trying to deliver gene therapy to patients with Duchenne muscular dystrophy. The company was originally founded in 1980 and trades on the NASDAQ under the ticker SRPT. Today its identity is built around the modern Sarepta of the 2010s onward, a company that has bet repeatedly that genetic medicine for neuromuscular diseases is worth pursuing even when the path through the FDA is hard.
The flagship product is Elevidys (delandistrogene moxeparvovec), the first gene therapy approved for Duchenne muscular dystrophy. Duchenne is a rare, progressive genetic disease that primarily affects boys, caused by mutations on the X chromosome. Patients are missing functional dystrophin, a protein their muscles need to repair themselves, so muscle tissue is gradually replaced by scar and fat over time. Most patients lose the ability to walk before adolescence and face premature death from cardiac and respiratory complications. Elevidys delivers a shortened working version of the dystrophin gene to muscle cells using a viral vector called AAV. The drug received FDA accelerated approval in 2023 for ambulatory boys aged 4 to 5, and the indication was expanded in 2024.
The Elevidys story has had a difficult more recent arc. In 2025, two non-ambulatory pediatric patients died from acute liver failure following Elevidys treatment. In November 2025 the FDA approved a Boxed Warning on the drug's label and revised the approved indication to limit use to ambulatory patients aged 4 and older. The FDA also required Sarepta to run an observational study of about 200 patients that checks liver function for at least 12 months after the infusion. For boys who can no longer walk, Sarepta is testing an added immune-suppressing medicine called sirolimus in Cohort 8 of its ENDEAVOR study, with full enrollment expected by the end of 2026. The events have made Elevidys a difficult and personal decision for many families weighing risk against the natural course of the disease, and Sarepta has been in active dialogue with the FDA and the DMD patient community since.
Sarepta also markets three earlier antisense oligonucleotide therapies for DMD that work by skipping over specific mutated exons of the dystrophin gene so a partially functional protein can still be produced. These are Exondys 51, Vyondys 53, and Amondys 45, each designed for a different DMDmutation type. In July 2025 Sarepta paused most of its limb-girdle muscular dystrophy gene therapies to cut costs, keeping SRP-9003 for LGMD type 2E/R4. That same month the FDA placed Sarepta's LGMD gene therapy trials, SRP-9003 among them, on clinical hold after a patient in one of its LGMD gene therapy trials died of acute liver failure. BioSpace reported on October 15, 2025 that the SRP-9003 hold was still in place, and Sarepta has not announced since then that it was lifted. Its pipeline now centers on siRNA medicines for facioscapulohumeral dystrophy, myotonic dystrophy type 1 and Huntington disease, with dosing underway in a first-in-human Huntington study.
Sarepta Therapeutics has active programs across 5 rare diseases: Duchenne Muscular Dystrophy, Facioscapulohumeral Muscular Dystrophy, Huntington Disease, Limb-Girdle Muscular Dystrophy, Myotonic Dystrophy. Each disease page on Trial Friend shows the latest recruiting trials, FDA-approved treatments, and patient resources.
Yes. Sarepta Therapeutics is a rare disease specialist headquartered in Cambridge, United States, founded in 1980. The company focuses primarily on developing treatments for rare and orphan diseases, with programs targeting Duchenne Muscular Dystrophy, Facioscapulohumeral Muscular Dystrophy, Huntington Disease among others.
The clinical trials section on this page shows active and recruiting rare disease trials sponsored by Sarepta Therapeutics, pulled live from ClinicalTrials.gov. Each trial card includes the study phase, recruitment status, conditions being studied, number of trial sites, and a direct link to the full study record. You can also explore individual disease pages to see trials from all sponsors for a specific condition.
To explore enrollment, find a recruiting trial in the clinical trials list on this page and click "View full study" to see the full ClinicalTrials.gov record, which includes eligibility criteria, study locations, and contact information for each trial site. Your doctor can also help determine if a specific trial is appropriate for your condition. Not all trials are open at every location, so check the site list for one near you.
Sarepta Therapeutics has 4 FDA-approved drugs with a rare disease use that we track, as of November 2026.
Source: the FDA's openFDA label database, matched to the rare diseases on Trial Friend; the list updates automatically and can include older products.
Yes, for 4 of its FDA-approved rare disease medicines that we track, each checked on Sarepta Therapeutics' own website.
Elevidys: SareptAssist, 1-888-727-3782. Offers copay help with private insurance and insurance help. Checked September 24, 2026.
Exondys 51: SareptAssist, 1-888-727-3782. Offers copay help with private insurance, a free medicine program and insurance help. Checked September 24, 2026.
Vyondys 53: SareptAssist, 1-888-727-3782. Offers copay help with private insurance, a free medicine program and insurance help. Checked September 24, 2026.
Amondys 45: SareptAssist, 1-888-727-3782. Offers copay help with private insurance, a free medicine program and insurance help. Checked September 24, 2026.
Medicare and Medicaid: drugmaker copay cards are limited to private insurance, so people with government coverage usually look to a free medicine program or a charity fund, both on the help paying page.
Eligibility is decided by each program, so call the number listed before a prescription is filled.
Sarepta Therapeutics was founded in 1980 and is headquartered in Cambridge, United States. The company has been active for 46 years. It trades publicly under the ticker symbol SRPT. As a rare disease specialist, the company dedicates its resources to developing therapies for patients with rare and orphan conditions.