News Related to Muscular Dystrophy Association

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Precision BioSciences Receives FDA Fast Track Designation for PBGENE-DMD Gene Editing Therapy

Mar 09, 2026

Precision BioSciences announced FDA Fast Track designation for PBGENE-DMD, a first-in-class gene editing therapy for Duchenne muscular dystrophy targeting mutations in exons 45-55, representing up to 60% of DMD patients.

Benitec to Present BB-301 Gene Therapy Results at March Conference

Feb 23, 2026

Benitec Biopharma will present interim clinical study results for BB-301 gene therapy in oculopharyngeal muscular dystrophy patients at the Muscular Dystrophy Association Conference on March 9, 2026, including 12-month and 24-month follow-up data.