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PPMD is proud to share an advance copy of Putting Patients First, a white paper outlining recommendations to speed responsible access to new therapies for Duchenne. Since we launched our advocacy efforts in 2000, we have seen continual progress in Washington. From the passing of the MD-CARE Act in 2001, to its reauthorization in 2008 (hopefully again in 2013), to historic PDUFA and FDASIA legislation passed July 2012. From virtually no funding to over $250 million leveraged in Duchenne-specific resources, the voices of this community continue to be heard in D.C.

But now it is time to take our discussions with federal regulatory agencies to the next level.

We have been working with the FDA and other regulatory agencies for the last decade to educate them on Duchenne and the catastrophic affect this disorder has on both patients and families. As our relationship continues to grow and build, and with the implementation of the FDASIA, we are hopeful that the FDA will work with PPMD and the rare disease community to take advantage of the new opportunities created by this landmark legislation.

To ensure we make the most of these new opportunities, PPMD brought together an expert Advisory Committee, including leading voices in academia, industry, and patient advocacy, to issue recommendations about how to effectively evaluate new therapies for Duchenne and other, rare, serious, and life-threatening disorders. We are pleased to share an advance copy of the recommendations with you. Read the white paper recommendations. For a brief synopsis of the white paper recommendations, see the executive summary.

After years of investments in research, there is now a very promising pipeline of therapies under investigation for the treatment of Duchenne. As you know, at least seven compounds are in Phase 2 or 3 clinical trials and are approaching consideration for approval. This rich pipeline is giving new hope to our community and we must work together to ensure that potentially life-saving therapies are evaluated in a manner that will allow us to speed responsible access.

We are optimistic that Putting Patients First will provide guidance for the FDA and other regulatory agencies as they continue to understand Duchenne and this community’s need for accelerated approval. This is an important document, vetted by top thought leaders in Duchenne and rare disease, and it is one of our proudest accomplishments. In the coming weeks and months we will be meeting with stakeholders and agency leaders to discuss the recommendations.


Be sure to check out our webinar on Putting Patients First – May 1st at 1 pm eastern.




Pat Furlong, Founding President, CEO
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