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Treatments on the Horizon
The Duchenne community is celebrating another promising step toward an approved treatment. On May 19 Sarepta Therapeutics announced plans to submit a rolling NDA for eteplirsen with the final […]
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 […]
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 […]
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 Phase 3 tadalafil DMD study: enrollment completed
CureDuchenne is pleased to share the following update to the Duchenne muscular dystrophy community from Lilly regarding the ongoing Phase 3 trial of tadalafil in boys with Duchenne. Eli Lilly […]
Keeping the Promise for our Children with Duchenne
Today, CureDuchenne announced a $1 million investment in Capricor Therapeutics for a clinical trial to test its CDC (adult) stem cells as a cardiac therapy for Duchenne patients. CureDuchenne’s mission […]
Sarepta Therapeutics Reports Long-Term Outcomes through 168 Weeks from Phase IIb Study of Eteplirsen in Duchenne Muscular Dystrophy
Sarepta Therapeutics released the 168 week data for their exon skipping drug, eteplirsen, this morning. During a three year period, 7-13 year old boys with Duchenne (the age range of […]
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 compound PF-06252616 in boys with Duchenne muscular dystrophy (DMD), a […]
TACT TREAT–NMD Review
Last weekend, I had the honor, along with Dr. Mike Kelly, CureDuchenne’s Chief Scientific Officer, and other TACT members, to review Duchenne research projects. CureDuchenne was able to look […]