Mendell 2015 - Phase 1/2a Follistatin Gene Therapy Trial for Becker Muscular Dystrophy

Molecular Therapy·January 1, 2015

Jerry R. Mendell

Summary

Follistatin gene therapy was evaluated as a myostatin-antagonist strategy in Becker muscular dystrophy, providing clinically relevant context for follistatin biology.

Study Details

Study Design

Phase 1/2a clinical gene-transfer trial

Indication

Becker muscular dystrophy

Intervention

AAV-mediated follistatin isoform gene delivery

Species

Human

Risk of Bias Assessment

Gene therapy study, not injectable Follistatin-344 peptide