Becker muscular dystrophy (gene therapy)
Mendell 2015 (Phase 1/2a, n=6) injected AAV1-FS344 into the quadriceps of Becker muscular dystrophy patients and reported some improvement in 6-minute walk distance and muscle fiber diameter. The trial is small, open-label, and uses gene therapy delivery — not exogenous recombinant peptide injection. Community use of synthetic 'follistatin-344 peptide' has no clinical evidence base.
1 human study · 8 animal studies
Key findings & citations
- Mendell 2015: AAV1-FS344 gene therapy improved 6MWT in some BMD patients.
- Kota 2009: AAV-FS344 increased muscle mass in nonhuman primates.
- All clinical work is gene therapy — not exogenous recombinant follistatin-344 peptide.
- No published human RCT of injected recombinant follistatin-344.
Our take
Gene therapy is not the same as injecting a peptide. Community use of 'follistatin-344' as a peptide injection has no clinical evidence — the published trials don't translate.