Sarepta Therapeutics, Inc.
Clinical trials sponsored by Sarepta Therapeutics, Inc., explained in plain language.
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New study aims to prevent liver damage from duchenne gene therapy
Disease control Not yet recruitingThis study tests whether adding the drug sirolimus can reduce the risk of acute liver injury in people with Duchenne muscular dystrophy who receive the gene therapy ELEVIDYS. About 20 participants will be enrolled to monitor safety and side effects. The goal is to make this treat…
Phase: PHASE4 • Sponsor: Sarepta Therapeutics, Inc. • Aim: Disease control
Last updated May 26, 2026 10:26 UTC
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New Huntington's drug enters first human safety trial
Disease control Not yet recruitingThis early-stage trial tests a new drug called SRP-1005 in 32 adults with Huntington's disease. The main goal is to check safety and side effects, not to cure the disease. Participants must have a confirmed genetic diagnosis and be in early to mild stages of the condition.
Phase: PHASE1 • Sponsor: Sarepta Therapeutics, Inc. • Aim: Disease control
Last updated May 22, 2026 13:53 UTC