We are pleased to report our final numbers for the FY19 letters.

In total, 19 Senators signed onto the Senate FY19 letter, joining 51 members of the House. That makes a solid 70 members of Congress (House and Senate combined) sending a strong message of support behind Duchenne research, public health, drug development, and patient support initiatives. They provide strategic direction for the federal agencies that touch Duchenne. Given the…

PPMD was excited to head to the Midwest for the second stop on the 2018 End Duchenne Tour. The event took place at Western Michigan University in Grand Rapids, Michigan, in partnership with Team Joseph, Little Hercules Foundations, and Noah’s Feat.…

Patient-Focused Drug Development in action

Companies developing gene therapies, the FDA, and other stakeholders are interested in the treatment priorities of the Duchenne community.  Because of this, PPMD has collaborated with RTI International, with financial support from Solid Biosciences and…

PPMD kicked off the 2018 End Duchenne Tour with a stop in our very own backyard - Goryeb Children's Hospital in Morristown, New Jersey! PPMD was extremely grateful to partner with Michael's Cause, Pietro's Fight, Powers Promise, and Ryan's Quest on…

The PPMD Advocacy Conference registration is live! Join us March 4-6 in Washington DC to advocate for Duchenne directly with Members of Congress.

Our…

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 and disease progression, and even where you live. With two…

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 organizations, a network of active families, and where most of the drug companies…

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…

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 did not release a more complete budget until the end of…

The newest version of the GOP HealthCare Bill is coming to a vote by 1:30 PM EDT this afternoon!

This is moving fast and we need ALL MEMBERS of our community to CALL your US…

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 PPMD’s Certified Duchenne…

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 Congress. We are delighted to report that the version in front…

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…

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 PPMD’s Certified Duchenne…

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 home through emails, phone calls, and social media – your actions…

Bipartisan Group of Senators Urge FDA to Utilize All Available Tools to Speed Access to Promising Duchenne Therapies

On Friday April 15th, U.S. Senators Roger Wicker, (R-MS) and Amy Klobuchar, (D-MN), sent a…

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), Bill Keating (D-MA), and Peter King (R-NY) led this incredible bipartisan letter.…

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 largest possible impact on the advisory committee and the…

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 supporting vital efforts that will move us closer to ending…

Thanks to this incredible community urging lawmakers, well over 100 Members of Congress signed and submitted a letter …