Due to overwhelming interest in PPMD’s Duchenne Policy Forum on December 12 in Silver Springs, MD, registration is now closed. We are at capacity and unfortunately there will be no onsite registration due to space restrictions.
Added by Ryan Fischer on December 6, 2013 at 11:30am — No Comments
With Congress back to work it is time we re-engage with members on moving the MD-CARE Act through the legislative process.
Where are we with the current bill?
Thanks to your efforts, we have collected …Continue
Added by Ryan Fischer on November 7, 2013 at 11:00am — No Comments
It’s that time of year again – the start of another school year. While we put our faith in schools to provide a proper, inclusive education for our children, faith is not all we have in our arsenal for ensuring this is the case for students living with Duchenne.
As parents, you are constant advocates for your children; this is especially true with…Continue
Added by Ryan Fischer on September 3, 2013 at 10:45am — No Comments
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…Continue
Added by Ryan Fischer on July 31, 2013 at 9:30am — No Comments
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
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
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…
In February 2013, a bipartisan group of Senators and Representatives introduced the MD-CARE Act Amendments of 2013 (S. 315 and H.R. 594). This legislation seeks to update the landmark MD-CARE Act law that has done so much over the past 12 years to extend and improve…Continue
Added by Ryan Fischer on May 29, 2013 at 11:30am — No Comments
A Call to Action for all NH Constituents!
Seacoast, New Hampshire- 8th grader Rebecca Gowing wanted to make a difference in her community. After learning that her sister’s classmate and friend, Lucas Currier, had Duchenne Muscular Dystrophy she took action to help raise funds and spread awareness, creating Art for A Cure…Continue
Added by Ryan Fischer on April 2, 2013 at 9:00pm — No Comments
Help Bring Run for Our Sons to your own backyard.
I hear it all the time, “I want to do a fundraiser but not sure what to do or where to start” – I ask “have you thought of doing a 5K or fun run in your community?” – The rest is Run for Our Sons history!
5Ks and fun runs are fast becoming one of PPMD’s…Continue
Added by Ryan Fischer on March 14, 2013 at 1:30pm — No Comments
Added by Ryan Fischer on March 7, 2013 at 4:21pm — No Comments
Updated 3/07/14: 109 total members- New additions in bold below
We continue to make progress on the Reauthorization of the MD-CARE Act. Thanks to you, we have…
Added by Ryan Fischer on March 5, 2013 at 3:00pm — No Comments
As you know, we head into an important year of Advocacy with the muscular dystrophy community determined to Reauthorize the MD-CARE Act. For the past few months PPMD, FED, and MDA have been working together on the legislation and coordinating efforts with our…Continue
Added by Ryan Fischer on January 28, 2013 at 4:30pm — No Comments
I am often asked: "How can I help in the fight to end Duchenne?" and "What are things I can do to make a difference?" My answer is always Advocacy.
You are already…Continue
Duchenne is getting a great deal of attention these days. With the excitement around the Sarepta trial and more trials coming online, more attention to Duchenne is a great thing as we continue to raise awareness. Personal stories about families only help to educate the public on Duchenne. It allows them to relate and perhaps to think, If this was my son, what would I do??
Added by Ryan Fischer on November 27, 2012 at 4:00pm — No Comments
Those engaged in federal advocacy probably thought the Prescription Drug User Fee Act and its acronym PDUFA would take the cake in terms of policy jargon. But as the nation's eyes are fixed on…
PPMD and other groups submit joint statement making the case for neuromuscular disorders affecting children
As many advocates know from earlier blog postings, PPMD has been deeply and passionately engaged to enhance how the Food and Drug Administration (FDA) goes about its business of reviewing potential therapies and treatments for patients with Duchenne. Our work is focusing on ensuring that the agency moves as expeditiously as possible and…Continue
Added by Ryan Fischer on November 15, 2012 at 11:00am — No Comments
Today, Parent Advocate and Founder of Two Smiles One Hope Foundation, Alison Willis and I represented the Duchenne community at the Congressional Rare Disease Caucus Briefing in Washington, DC. Alison did a remarkable job speaking on behalf of PPMD regarding the Patient Participation in Medical Product Discussions Provision within the newly passed…Continue
Added by Ryan Fischer on November 14, 2012 at 3:00pm — No Comments
While many of us – particularly those of you who live in key "swing" states that will likely determine the outcome of the election – may be tired of the unceasing television commercials, robocalls and mailers, it's important to recognize that regardless of who is elected, creating…Continue
Added by Ryan Fischer on November 5, 2012 at 2:30pm — No Comments
With the recent news of Sarepta's promising 48-week data, many have asked how accelerated approval may play into the FDA review of eteplirsen. Let's first define what accelerated approval means.