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
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.
Top citations
Mendell 2015 — BMD gene therapy Phase 1/2a
Kota 2009 — AAV-FS344 NHP
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.