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Hope and Gratitude: Moving Closer to a Cure for Duchenne
As I type this, my heart is filled with hope. A horizon that we could barely see 12 years ago has arrived. When our son Hawken was diagnosed with Duchenne […]
BioMarin Completes Rolling NDA Submission to FDA for Drisapersen for Treatment of Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
The PJ Nicholoff Steroid Protocol
CureDuchenne Cares is pleased to share important information about steroids use for Duchenne patients. Use of corticosteroids is part of the standards of care for Duchenne patients. Chronic use […]
Capricor Granted FDA Orphan Drug Designation for Allogeneic Cardiosphere-Derived Cells for the Treatment of Duchenne Muscular Dystrophy
Capricor Therapeutics, Inc. (Nasdaq:CAPR), a biotechnology company focused on developing novel therapeutics for the treatment of cardiovascular diseases, today announced that it has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA) for its cell therapeutic product candidate, CAP-1002, for the treatment of cardiomyopathy associated with Duchenne muscular dystrophy (DMD).
BioMarin Update on Duchenne Muscular Dystrophy Program
CureDuchenne is pleased to share the following communication update from BioMarin to the Duchenne community regarding developments of their exon skipping program for exon 51. CureDuchenne has provided extensive support […]
Update on Duplication 2 Research for Duchenne Muscular Dystrophy: Webinar with Dr. Kevin Flanigan
On April 6, 2015, CureDuchenne and Dr. Kevin Flanigan held a webinar to provide an update on Duplication 2 Research for Duchenne Muscular Dystrophy. Click here to watch the recording of the […]
In Silico Screening Based on Predictive Algorithms as a Design Tool for Exon Skipping Oligonucleotides in Duchenne Muscular Dystrophy
Clinical phenotypes as predictors of the outcome of skipping around DMD exon 45.