Mendell 2015 — Follistatin Gene Therapy Trial for Becker Muscular Dystrophy

Molecular Therapy

Jerry R. Mendell, et al.

Summary

Follistatin-344 gene transfer showed proof-of-principle safety and functional signals in Becker muscular dystrophy.

Study Details

Study Design

Phase 1/2a clinical gene-therapy trial

Indication

Becker muscular dystrophy

Intervention

AAV1.CMV.huFollistatin344 gene transfer

Species

Human

Sample Size

6 subjects

Risk of Bias Assessment

Open-label small gene-therapy trial; not injectable peptide use