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Speak Up for the Duchenne Community!
After waiting for a very long time, the Duchenne community now has clarity on the Advisory Committee meetings dates for BioMarin and Sarepta. BioMarin’s date is November 24, 2015 […]
Researchers Create New Mouse Model for Duchenne with Duplication of Exon 2
Dr. Kevin Flanigan and his team at the Flanigan Lab at Nationwide Children’s Hospital are hard at work researching duplication mutations. They have created a new mouse model for […]
Welcome Dr. Jak Knowles
We couldn’t be prouder that CureDuchenne has played a critical role in the development of what we hope will be the first-ever pharmaceutical treatments for Duchenne muscular dystrophy. Investments by […]
Do You kNOW Your Duchenne?
When our son, Hawken was diagnosed with Duchenne almost 13 years ago, my husband and I felt there couldn’t be too many things worse for a family to experience. […]
Duchenne Community: Let Your Voice Be Heard!
This week the FDA accepted Sarepta Therapeutics’ New Drug Application for eteplirsen with priority review. This follows on the June FDA’s acceptance of BioMarin’s NDA for drisapersen, also with […]
CureDuchenne’s Early Funding Projects Take a Big Step Forward Today
FDA Accepts NDA for Drisapersen, the First-Ever Treatment for Duchenne Muscular Dystrophy to Reach this Milestone in the U.S., and Sarepta Therapeutics Completes NDA Submission to FDA for Eteplirsen […]
Capricor Therapeutics – Webinar on Upcoming Clinical Trial for Cardiac Indication in Duchenne
There has been a lot of recent Duchenne research news lately. As a community, we are glad to see a variety of research programs moving forward with positive results. […]
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 […]