Early data show follistatin gene transfer may be safe and effective in BMD and safe in IBM, in which efficacy has not yet been evaluated
Preliminary results from a trial to test the safety of injecting follistatin genes into the thigh muscles of adults with Becker muscular dystrophy (BMD)or sporadic (nongenetic) inclusion-body myositis (sIBM) suggest that the experimental therapeutic approach is safe in both types of patients
“I was always interested in science, and specifically in science as it applies to medicine,” says Se-Jin Lee, a professor in the Department of Molecular Biology and Genetics at Johns Hopkins University School of Medicine in Baltimore.
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 May 1997, Kathryn Wagner was doing something she hadn’t had much time to do in years: She was watching television. Wagner had just given birth to her first baby, James, and was on leave from her postgraduate training in neurology at Johns Hopkins University School of Medicine in Baltimore.
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.
Brief items about myostatin inhibition, exon skipping and stem cell research in Duchenne muscular dystrophy
Anti-myostatin drug trial shows good preliminary results
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).