Navigating access to approved therapies can be both time consuming and daunting. The time between approval by FDA to the day of a patient’s first commercial dose can vary depending on which insurance you have, your age…Continue
Added by Ryan Fischer on November 20, 2017 at 8:00pm — No Comments
Boston made for a great location for our October Every Single One Tour stop. Nothing beats the northeast in the fall. Boston is home to a number of great Duchenne…Continue
Added by Ryan Fischer on October 30, 2017 at 2:00pm — No Comments
Navigating life with Duchenne can be daunting and time consuming for families. We rely on each other to share wisdom and information in order to make the process smoother and life a bit easier. The best guidance we can get is from those who have been through a given situation, sharing what they’ve learned to help others navigate resources that exist.
PPMD strives to find…Continue
Added by Ryan Fischer on June 21, 2017 at 10:00am — No Comments
With Congress not wrapping up spending bills for Fiscal Year 2017 until we were more than 7 months into the fiscal year, the FY 2018 budget and appropriations (funding) cycle will be interesting to say the least. The Trump Administration released a preliminary — or “skinny” budget -- in March but…Continue
Added by Ryan Fischer on June 15, 2017 at 11:30am — No Comments
The newest version of the GOP HealthCare Bill is coming to a vote by 1:30 PM EDT this afternoon!Continue
Added by Ryan Fischer on May 4, 2017 at 12:00pm — No Comments
Duke University marked our final stop on PPMD’s 2016 Every Single One Tour, held on November 2. We were thrilled to be holding another tour stop at one of…Continue
Added by Ryan Fischer on December 7, 2016 at 11:04am — No Comments
We need you to call your two US Senators TODAY with one simple message – VOTE YES ON 21st CENTURY CURES!!
With just days remaining in the current Congress, this bill represents the extraordinary efforts of our PPMD community’s work and outreach with…Continue
Added by Ryan Fischer on December 5, 2016 at 10:07am — No Comments
You are invited to join us February 12-14, 2017 in Washington D.C. for what is going to be another critical year in Duchenne advocacy.
2016 marked a tremendous…Continue
Added by Ryan Fischer on October 20, 2016 at 1:00pm — No Comments
This past weekend marked the third and fourth stops on PPMD’s Every Single [One] Tour – one taking place at Denver Children’s (University of Colorado) – one of…Continue
Added by Ryan Fischer on August 23, 2016 at 12:00pm — No Comments
Thanks to your advocacy, this week we learned that all of the Duchenne priorities we advocated for this year with Members of Congress are one step closer to being included in next year’s budget.
Whether you traveled to DC for face-to-face meetings with members, or advocated from…Continue
Added by Ryan Fischer on June 10, 2016 at 12:00pm — No Comments
Bipartisan Group of Senators Urge FDA to Utilize All Available Tools to Speed Access to Promising Duchenne Therapies
On Friday April…Continue
Added by Ryan Fischer on April 18, 2016 at 9:44am — No Comments
Earlier this year, 109 members of the House of Representatives sent a powerful letter to the FDA urging them to “utilize all available tools, resources, and authorities” in reviewing a treatment for Duchenne muscular dystrophy. Congressional champions Congressmen Mike Fitzpatrick (R-PA),…Continue
Added by Ryan Fischer on April 5, 2016 at 11:30am — No Comments
We are just a few short weeks from the FDA Advisory Committee Meeting for eteplirsen on April 25th. PPMD is part of a united coalition of Duchenne organizations (MakeDuchenneHistory Coalition) working together to ensure we make the…Continue
Urge your Lawmakers to Sign the FY 17 Duchenne Appropriations Letter!
Today and tomorrow, scores of PPMD advocates will be on Capitol Hill to urge their Senators and Members of Congress to…Continue
Added by Ryan Fischer on February 22, 2016 at 1:43pm — No Comments
Thanks to this incredible community urging lawmakers, well over 100 Members of Congress signed and submitted a letter …
PPMD and a coalition of Duchenne foundations continue to work together on preparing for the Sarepta Ad Comm. As of today, we are still waiting on a new confirmed date. With the postponement of the Ad Comm, the Congressional letter to FDA remains open. Has your House Members signed on…Continue
Added by Ryan Fischer on January 25, 2016 at 11:30am — No Comments
There has never been a moment like this in our Duchenne community. Today the FDA released briefing documents on the application for eteplirsen. It is clear that we continue to face a serious regulatory challenge as a community.…
In October, PPMD held a FACES Roundtable in Houston that included an incredible line up of global experts in care and research. In an effort to ensure this information reached families beyond Houston, we recorded the sessions. Special thanks to Rachel and James Poysky for pulling together such a…Continue
Added by Ryan Fischer on December 10, 2015 at 3:30pm — No Comments
Come and advocate for Duchenne directly with members of Congress.
PPMD's Annual Advocacy Conference, held in the first quarter of each year, is the only officially organized gathering on Capitol Hill for the Duchenne community. Families…Continue
Added by Ryan Fischer on December 3, 2015 at 10:54am — No Comments
We are thrilled to report that last night the Ensuring Access to Clinical Trials Act of 2015 (EACT) passed a vote in the House of Representatives (after passing the Senate by unanimous consent in July) and will go to the President for his signature!
This a result of everyone…Continue
Added by Ryan Fischer on September 29, 2015 at 11:08am — No Comments