MDA's 2015 Scientific Conference points the way toward tomorrow's therapies
Last month, MDA brought together more than 400 of the nation’s leading scientists, researchers and professionals specializing in neuromuscular disease research and care at its 2015 Scientific Conference in Washington, D.C., March 11–14. This pre-eminent gathering, hosted every other year, was designed to accelerate discoveries and drug development for families fighting neuromuscular diseases.
Research updates and clinical trials information
What About My Disease?
Readers sometimes wonder what’s happening with research for their diseases when they don’t see news about them for a while in the pages of Quest. But keep in mind: Research that seems to be for one disorder often has spillover implications for others.
Santhera Pharmaceuticals' investigational drug to treat respiratory dysfunction in Duchenne muscular dystrophy has shown benefit compared to a placebo in a phase 3 trial
Results from a phase 3, 65-participant trial of idebenone (brand names are Catena and Raxone) in boys with Duchenne muscular dystrophy (DMD) show the drug reduced the decline in respiratory function compared to a placebo, potentially paving the way toward regulatory approval.
In a May 22, 2014, letter, Prosensa provides updates on drisapersen and other Duchenne MD compounds in its pipeline, and on its natural history study
Dutch biopharmaceutical company Prosensa, developer of the experimental Duchenne muscular dystrophy (DMD) drug drisapersen, says it is moving the compound forward through regulatory agencies in the U.S. and Europe and will resume administering it to a first group of trial participants in the third quarter of 2014.
In its Summer 2013 round of grants, MDA awarded 31 new grants totaling $8.5 million.
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ALS (amyotrophic lateral sclerosis)
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