Featured in this issue: First U.S. exon-skipping trial opens * Utah researchers seek families with Ullrich CMD or Bethlem myopathy * Acceleron receives Fast Track, Orphan Drug designations for ACE031 * BioMarin abandons BMN195 development
Featured in this issue: Tadalafil tested in men with BMD * Tests of ACE031 in boys with DMD begin * New tools to measure progression of CMT in children * Idebenone posts disappointing results in FA
For information about active trials in your disease, go to www.clinicaltrials.gov, select Search for Clinical Trials, and enter the disease name in the search box.
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).
This article contains items about: Pompe disease (acid maltase deficiency), Lambert-Eaton myasthenic syndrome, Duchenne and Becker muscular dystrophies, mitochondrial myopathy, myasthenia gravis and spinal muscular atrophy
A report on drug development in neuromuscular diseases as of January 2009
In the era of molecular biology, the drug development process has moved from a “let’s try it and see what happens” approach to a scientifically based process of discovery and application.
For many of the diseases in MDA’s program, drug discovery begins with gene discovery — identifying a gene that, when flawed, causes a disease.