Clinical Trials and Studies
Jim Rusche from Repligen Corp. announced encouraging interim results from a phase 1 clinical trial of RG2833 in Friedreich's ataxia
PTC Therapeutics' large-scale multinational trial of ataluren for nonsense-mutation Duchenne or Becker MD has opened its first site in Cincinnati, Ohio
Isis Pharmaceuticals has launched a phase 2 trial to test its experimental antisense drug ISIS-SMNRx in infants with spinal muscular atrophy
Researchers conducting a study of medical changes and complications in myotubular myopathy (MTM) are seeking individuals with MTM or their parents for 12 monthly phone interviews
In early 2011, clinical trials of ACE-031, a myostatin inhibitor, were halted pending resolution of safety issues; in May 2013, the developers discontinued work on the drug
A study seeks people suspected of having myotubular myopathy who have not yet had their MTM1 gene mutation confirmed by a clinically certified lab
FDA has asked Sarepta Therapeutics for additional information about dystrophin production and safety before it will consider accepting an application for accelerated approval of its exon-skipping drug
Weekly injections of the exon-skipping drug drisapersen in boys with Duchenne muscular dystrophy resulted in a significant difference in walking distance compared to a placebo
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