PTC Therapeutics: Letter to the DMD Community
PTC Therapeutics President and CEO Stuart Peltz, Ph.D., issued a letter to the DMD community after the FDA declined to approve ataluren (brand name Translarna) for the treatment of DMD.
Tag: Research Advance
FDA Approves Emflaza for Treatment of Duchenne Muscular Dystrophy
The U.S. Food and Drug Administration has granted approval for deflazacort (brand name Emflaza), under development by Marathon Pharmaceuticals, to treat DMD
Recent Treatment Triumphs Tied to MDA’s Long Commitment to Funding Treatments and Cures
Over the last six decades, MDA has committed $1 billion to research designed to move the world closer to treatments and cures for muscular dystrophy, ALS and related life-threatening diseases. As a result, MDA’s fingerprints are on nearly every major neuromuscular disease research breakthrough, and the progress we continue to see in the field is . . .
