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FDA Advisory Committee Meeting
We are looking forward to the U.S. Food & Drug Administration (FDA) Advisory Committee meeting on Tuesday, November 24. This will be an important day for the Duchenne community. […]
BioMarin Quarterly Update to the Duchenne Community
CureDuchenne is pleased to share the BioMarin Quarterly Update to the Duchenne Community. It covers important information about next week’s FDA Advisory Committee meeting and other key updates from […]
You are Pioneers Once Again
We are pleased to share the following letter from the CEO of PTC Therapeutics to the Duchenne community. CureDuchenne funded PTC Therapeutics back in 2003 and we are proud […]
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 […]
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 […]
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 […]
FDA Webinar on Accelerated Approval Summary
CureDuchenne was honored to host a webinar today, in collaboration with PPMD and MDA to discuss accelerated approval for drugs for rare diseases. Dr. Robert Temple, director of the […]
CureDuchenne – Clinical Trials – FDA
Following the long awaited news of Sarepta’s phase IIB results for eteplirsen and continued Phase III progress of GSK/Prosensa’s drisapersen, as well as the recent developments with PTC Therapeutics and […]