Efmitermant alfa (ACE-083), a follistatin-based fusion protein with localized action, functions as a muscle-promoting agent and is utilized in the study of muscle disorders [1] [2].

ACE-083 (0-60 nM) exhibits high affinity for heparin and the extracellular matrix [1].

ACE-083 (muscle injection; 0-100 µg; biweekly for 4 weeks) induced local, dose-dependent hypertrophy in the injected muscles of both wild-type mice and mouse models of Charcot-Marie-Tooth disease (CMT) and Duchenne muscular dystrophy [1]. Moreover, the compound enhanced in situ isometric contraction force in the tibialis anterior muscle of treated wild-type and disease model mice, and increased dorsiflexion torque at the ankle joint in CMT mice [1].