Tag: Duchenne

A total of 13 posts are filed under Duchenne
PTC Announces Results from Phase 3 ACT DMD Clinical Trial of TranslarnaTM (ataluren) in Patients with Duchenne Muscular Dystrophy
PTC Therapeutics, Inc. recently announced results from the Phase 3, double-blind, placebo-controlled, 48-week ACT DMD trial of TranslarnaTM… Continue Reading
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Biomarin Launches kNOWyourDuchenne Program Offering Genetic Testing
BioMarin has officially launched kNOWyourDuchenne, a program dedicated to assisting families and clinicians gain access to genetic testing… Continue Reading
Struggling to Run Around on the Playground, 9-Year-Old Recieves Startling Diagnosis
Michael Capolongo at the age of 4 was complaining of leg pain when running around the playground with his pre K classmates. His pediatrician… Continue Reading
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Duchenne Community Finds Hope in Anaheim Ducks Captain Ryan Getzlaf and Hawken Miller
As Ryan Getzlaf loads a baby seat into the trunk of his white Mercedes S63, a pack of young fans swarms him in hopes of an autograph, a… Continue Reading
Pfizer Initiates Phase 2 Study of PF-06252616 in Duchenne Muscular Dystrophy
Pfizer Inc. (NYSE:PFE) announced today enrollment of the first patient in a multicenter Phase II clinical trial of the investigational… Continue Reading
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Parent Project Muscular Dystrophy Names Baltimore’s Kennedy Krieger Institute a Certified Duchenne Care Center
 Kennedy Krieger Institute in Baltimore, Maryland was named a Certified Duchenne Care Center by Parent Project Muscular Dystrophy… Continue Reading
Duchenne Muscular Dystrophy Experiment in Dogs Restores Key Dystrophin Levels
A study entitled “Forelimb Treatment in a Large Cohort of Dystrophic Dogs Supports Delivery of a Recombinant AAV for Exon Skipping in… Continue Reading