The small human FS344 study used an AAV gene-transfer vector in Becker muscular dystrophy. It was uncontrolled and cannot validate benefits, exposure or safety of injected follistatin-344 protein sold for research.[1]
- Gene therapy and administered protein differ fundamentally in formulation and duration of expression.
- A targeted follow-up literature search (Europe PMC, 2026-09-09) did not identify a dedicated later human follow-up trial of this six-patient cohort or a placebo-controlled confirmatory study within the top results screened.
Inspection history
Clinical review: not reviewed by a qualified clinician.
· Independently inspected the listed passages; replaced or qualified inherited assertions. No qualified clinical review has occurred.
· Independent fresh reinspection for the deep-evidence pass: reopened every cited source at its passages via NCBI E-utilities / DailyMed / accessdata.fda.gov / govinfo.gov / Wayback Machine mirror, checked Europe PMC correction/retraction metadata where applicable, and confirmed the claim's wording against the primary text. Disposition: supported within the stated scope. No qualified clinical review.