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ACE083

ACE083, ACE-083, ACE 083

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ACE-083 was an investigational drug (follistatin-based protein therapeutic) developed by Acceleron Pharma to treat muscle-wasting diseases like Charcot-Marie-Tooth (CMT) disease and facioscapulohumeral muscular dystrophy (FSHD). Its development was discontinued due to a lack of functional improvement in patients during Phase 2 clinical trials, despite increasing muscle mass. 
  • Mechanism of Action: ACE-083 was a locally acting “ligand trap” designed to inhibit specific proteins in the transforming growth factor-beta (TGF-β) superfamily, such as myostatin and activin, which naturally limit muscle growth. By binding to these proteins, the drug was intended to increase muscle mass and strength specifically in the injected muscle without systemic effects.
  • Administration: The therapeutic was administered via injection directly into the affected muscle (e.g., the tibialis anterior muscle in the lower leg or the biceps brachii in the upper arm).

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