ACE-083 is an investigational drug containing a modified form of human follistatin that can potentially increase muscle growth in a wide range of neuromuscular disorders. The compound has been studied in Phase 2 clinical trials to evaluate its safety, tolerability, pharmacodynamics, efficacy, and pharmacokinetics.
The study aimed to increase muscle volume and improve functional outcomes in adults with FSHD, a slowly progressive muscular dystrophy. The results showed that ACE-083 significantly increased total muscle volume but showed no consistent improvement in applicable or PRO measures in either group.
Pharmacokinetics studies how a drug is absorbed, distributed, metabolized, and eliminated by the body. However, recent clinical trials for Charcot-Marie-Tooth disease have failed to demonstrate functional improvement compared to a placebo, leading to the discontinuation of the drug’s development by Acceleron Pharma Inc.
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