UPDATED 2/11/14

As we move into the start of 2014 and the February Advocacy Conference we are hopeful to gain momentum to move MD-CARE Act to committee action this year. With 78 cosponsors in the House and 17 in the Senate, we need your help to up our numbers in both chambers. Target states in both the Senate…

In 2008, PPMD joined the Genetic Alliance and others in the rare disease community, in support of “open access” – essentially ensuring that all NIH funded researchers make available to the public published peer-reviewed manuscripts and articles on a searchable website like PubMed Central or the National Library of Medicines. Making them freely accessible to the public within 12 months of publication.

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A big day for Duchenne – Take action to help make it even more impactful!

I had the pleasure to join other Duchenne advocates in Washington this week in an effort to bring attention to the need to accelerate treatments for Duchenne, highlighting eteplirsen as a prime example of a promising potential treatment for a subset of the Duchenne population.

A briefing was held at the Rayburn House Office Building, sponsored by Congressman Keating and…

Tomorrow, Tuesday July 9^th, is a big day for Duchenne advocacy. Advocates will be speaking about the high unmet medical need and urgency for treatments from the halls of Congress to the offices of the FDA.



PPMD will be meeting with individuals from FDA to include CDER, Division of Neurology, Office of Strategic Programs and Staff from the Office of…