Lend Your Voice to the FDA Draft Guidance on Duchenne to Speed the Development of Therapies

Strength Happens Together

For the past four months, over 80 experts in Duchenne (including patient representatives and groups, industry, clinicians, and researchers) have been working hard to produce draft guidance on Duchenne that we believe will ultimately speed the development of therapies. 


PPMD is proud to announce the posting of a first pass of the draft guidance, entitled Duchenne Muscular Dystrophy: Developing Drugs for Treatments over the Spectrum of Disease.  

Now we want to hear from you, the Duchenne community, about your thoughts on the guidance content. Please submit your comments via PPMD’s website by Tuesday, June 10thAll comments will be sent on to the Steering Committee. 

The purpose of this guidance is to assist sponsors in the clinical development of medical products for the treatment of Duchenne over the entire spectrum of the disease (ambulatory and non ambulatory). It is the result of the first collaboration between the FDA and a rare disease community to produce clinical guidance in their respective disease area.

We are excited to finalize the guidance and send it to the FDA in the coming weeks. We also want to thank all the families who participated in our "Tell Your Story to the FDA" project. We are sending the agency ALL submitted stories for their consideration when making decisions about potential therapies. The stories are heartfelt, honest, and convey the need for urgency for accelerating approvals for our community. 

We hope you’ll take advantage of this opportunity to provide feedback to this historical document. We want to make sure we provide the FDA with a guidance that truly reflects the concerns and priorities of the entire Duchenne community.

Read the first pass & submit your comments.

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