Registry and studies open to people with CMT
When John Knopf co-founded Acceleron Pharma in the spring of 2003, a muscle protein called myostatin had been on his and other researchers’ radar for several years.
The protein had been identified as a “negative regulator” (limiter) of muscle growth and regeneration back in 1997. And, since that time, myostatin-deficient mice and cattle had been shown to have large, strong muscles without...
In this issue: Acceleron trial of ACE-031 in DMD * Santhera trial of idebenone in DMD * NIH seeking parents’ perceptions of DMD/BMD ataluren trials * Becker MD study seeks participants * Biobank collecting blood samples for neuromuscular disease research * Taiwanese trial finds hydroxyurea ineffective in types 2 and 3 SMA
For current information on clinical trials and studies, see the database at...
MDA has awarded $1.5 million to Acceleron Pharma for continued testing of ACE-031, an inhibitor of myostatin and related proteins, in children with Duchenne MD
MDA has begun funding tests of the experimental drug ACE-031 in children with Duchenne muscular dystrophy (DMD). The drug is being developed by Acceleron Pharma, a Cambridge, Mass., biotechnology company in collaboration with Shire, a global specialty biopharmaceutical company that focuses on developing, manufacturing and commercializing therapies for rare genetic diseases.
Tests show the myostatin inhibitor ACE031 can safely increase muscle size, a finding that could benefit many forms of neuromuscular disease
ACE031, a laboratory-modified protein developed by Acceleron Pharma of Cambridge, Mass., has shown promise as a therapy to increase muscle mass, based on results of a trial in healthy volunteers. The company will now test it in Duchenne muscular dystrophy (DMD).