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 in duchenne gene therapy
Disease control Not yet recruitingThis study tests whether adding sirolimus to ELEVIDYS treatment can prevent serious liver injury in boys with Duchenne muscular dystrophy. About 20 participants will receive the drug combination and be monitored for side effects. The goal is to make an existing gene therapy safer…
Phase: PHASE4 • Sponsor: Sarepta Therapeutics, Inc. • Aim: Disease control
Last updated May 14, 2026 12:06 UTC
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New Huntington's drug enters first human safety trial
Disease control Not yet recruitingThis early-stage study 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 will receive either the drug or a placebo, and researchers will measure how the drug moves thro…
Phase: PHASE1 • Sponsor: Sarepta Therapeutics, Inc. • Aim: Disease control
Last updated May 06, 2026 16:15 UTC