Sarepta Therapeutics, Inc.
Clinical trials sponsored by Sarepta Therapeutics, Inc., explained in plain language.
A biopharmaceutical company focused on RNA-targeted therapies for rare diseases.
Not investment advice. Company information is shown for context only. Clinical trials frequently fail.
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Upcoming readouts
Trials from this sponsor expected to report results within the next 12 months.
- Higher dose of muscular dystrophy drug shows promise in new trial Results expected Oct 2026 (estimated)
- New hope for muscle disease: experimental drug ARO-DM1 enters human trials Results expected Dec 2026 (estimated)
- Gene therapy breakthrough? new trial targets duchenne muscular dystrophy Results expected May 2027 (estimated)
- New study aims to make duchenne gene therapy safer for kids Results expected Aug 2027 (estimated)
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New gene therapy trial hopes to restore muscle protein in duchenne boys
Disease control Recruiting nowThis study tests a one-time gene therapy called delandistrogene moxeparvovec in 83 people with Duchenne muscular dystrophy. The goal is to see if it is safe and helps the body make dystrophin, a protein missing in Duchenne. The trial is now enrolling non-ambulatory participants (…
Phase 1 • Sponsor: Sarepta Therapeutics, Inc. • Aim: Disease control
Last updated Sep 13, 2026 00:00 UTC
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New drug SRP-1005 takes first step against Huntington's
Disease control Recruiting nowThis study tests a new drug called SRP-1005 in 32 adults with Huntington's disease. It is the first time this drug is being tested in humans, so the main goal is to check safety and how the body processes the drug. Participants will receive either SRP-1005 or a placebo by injecti…
Phase 1 • Sponsor: Sarepta Therapeutics, Inc. • Aim: Disease control
Last updated Sep 06, 2026 00:00 UTC
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New hope for muscle disease: experimental drug ARO-DM1 enters human trials
Disease control Recruiting nowThis study tests a new drug called ARO-DM1 in 78 adults aged 18-65 with type 1 myotonic dystrophy, a genetic muscle disorder. The goal is to check safety and how the body processes the drug. Participants receive either ARO-DM1 or a placebo by IV or injection. It is an early-stage…
Phase 1/2 • Sponsor: Sarepta Therapeutics, Inc. • Aim: Disease control
Last updated Aug 13, 2026 00:00 UTC
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New hope for rare muscle disease: experimental drug enters human trials
Disease control Recruiting nowThis study tests an experimental drug called ARO-DUX4 in people with facioscapulohumeral muscular dystrophy type 1 (FSHD1), a genetic condition that causes muscle weakness. The main goal is to check the drug's safety and how the body processes it. About 60 adults and teens will r…
Phase 1/2 • Sponsor: Sarepta Therapeutics, Inc. • Aim: Disease control
Last updated Aug 13, 2026 00:00 UTC