Drug Name: ACE-031
Company: Shire
Location: US-Massachusetts
Drug Type: Protein Biologic
Conditions: ALS, Duchenne muscular dystrophy
Mechanism Type: Improving muscle function
Mechanism: ACE-031 is a monoclonal antibody that binds and inhibits myostatin. It acts as a decoy receptor for myostatin and other proteins that limit muscle growth. Myostatin blockage has been considered as a therapeutic approach for ALS by improving muscle size and strength.
U.S. Status for ALS: Discontinued

[1] Myostatin inhibition slows muscle atrophy in rodent models of amyotrophic lateral sclerosis. Holzbaur, EL et al. Neurobiol Dis. 2006 Sep;23(3):697-707.
[2] Study of ACE-031 in Subjects With Duchenne Muscular Dystrophy., 31 Jan 2013. Accessed 11 Mar 2016 from
[3] UPDATE: ACE-031 clinical trials in Duchenne MD Wahl, M. Quest Magazine Online, 2 May 2013. Accessed 9 Mar 2016 from

Last updated March 11th, 2016

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