Sarepta Announces First Clinical Data from siRNA Pipeline Targeting FSHD1

U.S. biopharmaceutical company Sarepta has announced preliminary trial results for “SRP-1001,” a potential treatment for FSHD1.

The drug is designed to inhibit DUX4, a protein implicated in the cause of FSHD, and has so far shown promising results regarding muscle delivery and safety.

However, further trials are needed to confirm its actual efficacy.

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https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-announces-first-clinical-data-sirna-pipeline-targeting