Reviews

Summary

Positives

  • Patients with sporadic inclusion-body myositis treated with AAV-delivered follistatin gene therapy showed measurable gains on the 6-minute walk test and slowed disease progression compared with natural-history controls PMC.

  • Becker muscular dystrophy participants in the same gene therapy trial regained ambulation distance after years of decline PMC.

Negatives

  • Some peptide users reported fatigue, headaches, and flu-like symptoms during high-dose follistatin-344 cycles PMC.

  • Because follistatin also binds activin and other TGF-beta ligands, patients risk off-target endocrine effects that have not been mapped in human dose-response studies PMC.

Hurdles & Side Effects

  • Subcutaneous follistatin-344 has no published human dose-response data, so patients with post-viral muscle wasting are essentially self-experimenting outside any clinical framework Nature Scientific Reports.

  • The robust clinical evidence comes from sustained gene-therapy expression, a fundamentally different exposure pattern than the brief peaks produced by injected peptide PMC.

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