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…

Today is a landmark day for the entire Duchenne community. Not too long ago, the Duchenne therapy landscape was a barren field with little to no life. Thanks to a commitment to our patients and the field by the U.S. government, international governments, the venture community and industry, this landscape has changed markedly to the point where today we have the announcement for a…

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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July 9, 2013.  

PPMD met with Dr. Janet Woodcock and leadership from the Office of New Drugs, Medical Policy and the Division of Neurology Products, as well as, staff from the Office of Strategic Programs, Office of Health and Constituent Affairs.   The purpose of the meeting was to continue our efforts to provide the agency relevant information and data about Duchenne that will encourage/enable them to be flexible in their review process.  

We…

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…