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…Continue
Added by Ryan Fischer on July 31, 2013 at 9:30am — No Comments
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…Continue
anyone have any experiences using steroids with their son??? My son is 5 and they r thinking it might b time...not sure how I feel?? help!!
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
For six years now, the Dumm family in North Royalton, Ohio has been hosting JOA Picnic in the Park to raise money and awareness for Duchenne research. This is just one of the many things John Owen's Adventure, Inc. (JOA) does for our community. At the heart of JOA is 9-year-old John Owen Dumm who is a rising fourth grader with Duchenne and an active participant in his namesake event. In fact, John Owen and JOA have…Continue
Added by Will Nolan on July 16, 2013 at 3:03pm — No Comments
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…Continue
Added by Ryan Fischer on July 12, 2013 at 10:57am — No Comments
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
What a great day to celebrate life and enjoy all the wonderful things life has to offer. I can sit here and cry and feel sorry for what the future holds for my son who has Duchenne's MD, and for all the family, but I realized that I need to wipe my tears and continue to fight for my son and give him all the love in the world. Who knows what the future holds? I do know that this is such a beautiful gift that God has given me and I will love him more every day.
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…
On July 9, PPMD will be meeting with individuals from FDA to include CDER, Division of Neurology, Office of Strategic Programs and Staff from the Office of health and Constituent Affairs.
The topics for discussion will include a review of PPMD’s Board Policy, developed in 2011. This policy embraces the following principles supporting an enhanced regulatory framework to advance development of drugs for rare diseases:
Added by Pat Furlong on July 3, 2013 at 1:30pm — No Comments