FDA Draft Guidance on Duchenne: Telebriefing


On June 25, 2014, we shared the first-ever rare disease patient-initiated guidance with FDA to help accelerate treatments for Duchenne. This new guidance focuses on overcoming challenges in clinical trial design across six key areas. It is our profound hope that this effort will lead to the approval of much needed treatments for all people living with Duchenne.

Download the recording of our telebriefing to learn more about the guidance, the 80+ expert stakeholders who contributed, and our next steps.

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