Sarepta shares early clinical data from siRNA treatments for rare diseases
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Sarepta Announces First Clinical Data from siRNA Pipeline Targeting FSHD1 and DM1
March 25, 2026 8:05 AM EDTIn early clinical results, Sareptas v6 integrin-targeted siRNA approach achieves high muscle concentrations without dose limiting toxicity for FSHD1 and DM1 Company to host investor call on March 25, 2026, at 8:30 a.m. Eastern time
CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Sarepta Therapeutics, Inc. (NASDAQ: SRPT), the leader in precision genetic medicine for rare diseases, today shared the first clinical results from two of its siRNA programs for neuromuscular diseases.
Early results from Phase 1/2 ascending dose studies of SRP-1001 for facioscapulohumeral muscular dystrophy type 1 (FSHD1) and... More
