Featured Blog Posts – July 2013 Archive (5)

MD-CARE Act: Is your member in a target state or district?

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…


Added by Ryan Fischer on July 31, 2013 at 9:30am — No Comments

Sarepta Therapeutics announces plans to submit New Drug Application to FDA for Eteplirsen – A significant step for the Duchenne community

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…


Added by Pat Furlong on July 24, 2013 at 11:00am — 1 Comment

Advocating for Open Access

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.



Added by Ryan Fischer on July 18, 2013 at 4:30pm — No Comments

7.9.13: Update from PPMD/FDA Meeting

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.  



Added by Pat Furlong on July 12, 2013 at 10:39am — No Comments

July 9, 2013: One Voice from Capitol Hill to the FDA

Tomorrow, Tuesday July 9th, 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…


Added by Ryan Fischer on July 8, 2013 at 10:54am — 3 Comments

Featured Monthly Archives











Need help using this community site? Visit Ning's Help Page.



© 2022   Created by PPMD.   Powered by

Badges  |  Report an Issue  |  Privacy Policy  |  Terms of Service