New study aims to make duchenne gene therapy safer for kids
NCT ID NCT07542314
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests whether adding sirolimus (a drug that calms the immune system) to ELEVIDYS gene therapy can prevent serious liver injury in boys with Duchenne muscular dystrophy. About 20 participants will receive the treatment and be monitored for side effects. The goal is to make an existing therapy safer, not to cure the disease.
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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
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The condition terms exactly as the trial's registrant entered them.
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