There are hardly words to capture the impact our unified community had on Monday. We had traveled from all over the nation to convene with federal policymakers, industry partners, and payers for the Duchenne Patient-Focused Compass Meeting: Navigating our Pathway Forward. Nested within PPMD’s…

Summit has announced additional positive data from its PhaseOut DMD clinical trial today, showing a significant reduction in muscle inflammation after 24 weeks of ezutromid dosing. The findings in their 24-week interim data are consistent with the expected…

Capricor Therapeutics announced the online publication in Stem Cell Reports of a new study by researchers at the Smidt Heart Institute at Cedars-Sinai Medical Center, who found that cardiosphere-derived cells (CDCs) improved cardiac muscle function, walking abilities and survival in a mouse model of Duchenne. The CDCs used in the study are the research grade version of CAP-1002,…

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As part of this year's Advocacy Conference (March 4-6, 2018), PPMD will host the Duchenne…

PTC Therapeutics, Inc. announced that the Office of New Drugs of the U.S. Food and Drug Administration has reiterated the FDA's prior position and denied PTC's appeal of the Complete Response Letter in relation to the New Drug Application (NDA) for ataluren. In its letter, the Office of New Drugs recommended a possible path forward for the ataluren NDA submission…

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…

Last week, Parent Project Muscular Dystrophy and Action Duchenne hosted a webinar update on the recent report that Summit has announced positive 24-week interim results from the open-label Phase 2 proof of…

Today is a historic moment for the Duchenne community, as moments ago the FDA published the finalized “Duchenne Muscular Dystrophy and Related Dystrophinopathies: Developing Drugs for Treatment” Guidance for Industry…

Catabasis Pharmaceuticals reported new positive efficacy and safety results showing preservation of muscle function and sustained disease-modifying effects in boys with Duchene in the MoveDMD trial open-label extension following 48 and 60 weeks of treatment with edasalonexent. Consistent improvements in all assessments of muscle function were observed after more than a year of oral 100 mg/kg/day edasalonexent treatment compared to the rates of change in the…

The Americans with Disabilities Act (ADA) is in jeopardy and a current bill in the House of Representatives threatens to undermine the protections of the ADA.

We need all members of our Duchenne community to contact your Representative today by calling the main Capitol line at: 202-224-3121…

UPDATE - FEBRUARY 20, 2018: Despite bold outcry from disability and civil rights groups, HR 620 has passed in the House. PPMD has been in touch with Senate leadership and the path in the Senate is uncertain, but we urge our community to…

Care for the heart has long been a priority for PPMD. Over the past several years, PPMD’s Cardiac Initiative has given rise to:

• A meeting convened in partnership with the National Heart Lung and Blood Institute that included standard…

Santhera Pharmaceuticals announced the launch of a U.S. Expanded Access Program (EAP) referred to as BreatheDMD with idebenone for people with Duchenne. Through the BreatheDMD program, eligible patients in the U.S. with Duchenne who are 10 years and older and in respiratory function decline, can obtain access to…

Today Santhera announced that they have received a negative opinion on the Appeal for Authorization of Raxone®…

Today, Summit announced positive 24-week interim data from PhaseOut DMD, their Phase 2 clinical trial of the utrophin modulator ezutromid.…

PPMD is thrilled to announce that after many years in development, the Centers for Disease Control (CDC) has published updated Care Considerations for Duchenne in The Lancet Neurology. These articles are also now…

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…

In January of 2014 we held our inaugural PAAC Leadership Workshop in Washington,DC. The day included engagement with federal policy makers, congressional staff, and research leaders. At the end of the day, a long-time colleague of mine from the FDA who had spent the entire day with us pulled me aside and said, “Wow. These men are brilliant. They have such…

(Photo used with permission from family.)

“DMD gene therapy went well. It was started at 1:15 PM and ended at 2:27 PM”    

On Thursday, January 4, I received this message from Dr. Jerry Mendell accompanied by the photo above – Dr. Mendell at…

Remember that hot June day in 2015 when hundreds of Duchenne community members rallied together at the U.S. Capitol for PPMD’s EndDuchenne rally? That same day, our longtime Congressional champions Senators Roger Wicker (R-MS) and Amy Klobuchar (D-MN) introduced the Patient Focused Impact Assessment Act (PFIA, S. 1597) — and our 200+ PPMD advocates…