Parent Project Muscular Dystrophy (PPMD)

PPMD Together is headed to Philly! Register now for our first PPMD Together event of 2026 — join us in Philadelphia, PA, on April 24-25 for a weekend of gratitude, connection, and progress! Learn more about the agenda, accommodations, and register today: donate.parentprojectmd.org/event/ppmd-tog…

📢 News: @Capricor has announced additional analyses and new functional outcomes data from the Phase 3 HOPE-3 clinical trial of Deramiocel in #Duchenne. Learn more: parentprojectmd.org/capricor-thera…

📢 News: @Solid_Bio has shared updated positive interim data from the ongoing Phase 1/2 INSPIRE DUCHENNE clinical trial of SGT-003. Learn more: parentprojectmd.org/solid-bioscien…

📢 News: @REGENXBIO has announced new positive interim data from the Phase I/II AFFINITY DUCHENNE trial of RGX-202. Learn more: parentprojectmd.org/regenxbio-shar…

Together with @DuchenneMD, we are pleased to announce @Troy_Balderson, @SenSusanCollins, @SenAmyKlobuchar, Congresswoman Doris Matsui, and @SenatorWicker as recipients of the 2026 Advocacy Leadership Awards! #PPMDAdvocacy parentprojectmd.org/ppmd-and-fed-a…

🏛️ 📣 PPMD’s 2026 Advocacy Conference kicks off TODAY! Here’s a quick rundown by the numbers: 📌 120+ Duchenne and Becker advocates 📌 Representing 31 states 📌 2 days of advocating on Capitol Hill 📌 134 Congressional meetings #EndDuchenne #PPMDAdvocacy

Joining PPMD in support of the eDNHS study is @Insmed; ITF Therapeutics ; @NSPharmaInc; @Santhera (Switzerland); @Sarepta; @Solid_Bio; @trinds_nmd; and @UCDavisHealth. Learn more join us 4/1/26 for a webinar: parentprojectmd.org/ppmd-partners-…

The eDNHS is an expanded continuation of the previous Duchenne Natural History Study, which ran from 2005-2016, and will facilitate development and approval of novel therapies and improve global clinical practice and policy guidelines for the #Duchenne community.

Exciting news! We are proud to share our partnership with the Cooperative International Neuromuscular Research Group (CINRG) to advance the expanded Duchenne Natural History Study (eDNHS) study.

We are disappointed to learn the news that @Roche has made the difficult decision to terminate their Phase 2 SHIELD DMD study evaluating satralizumab for bone health in Duchenne. Read more about this decision: parentprojectmd.org/roche-announce…

@PTC announced their decision to withdraw their submission for Translarna™ (ataluren). This decision is based on recent discussions with FDA. Read a Message from PPMD’s President, Pat Furlong here: parentprojectmd.org/ptc-therapeuti…

@SatellosBio announces dosing of the first participant in the Phase 2 trial of SAT-3247, an investigational therapy being studied as a novel treatment to regenerate skeletal muscle for individuals with Duchenne. Read the press release to learn more: ir.satellos.com/news/news-deta…

The hotel reservation deadline for PPMD’s 2026 Advocacy Conference is extended to Tuesday, February 17! Don’t miss your chance to join us March 8-10th in #DC to advocate for the Duchenne and Becker community in the nation’s capital!

✨ Have you registered for PPMD’s 2026 Annual Conference? We invite you to join us in Orlando, June 25-27, for a little magic. Book your stay through PPMD’s hotel block at Disney’s Coronado Springs Resort! And be sure to register by 5/14 for best rates. parentprojectmd.org/conference

❗BREAKING NEWS WEBINAR: Join PPMD and Sarepta for a breaking news community webinar TODAY, Monday, January 26, at 4:00 PM ET to discuss these topline results and answer questions from the community. parentprojectmd.org/sarepta-shares…

Today @Sarepta announced positive topline three-year functional results from Part 1-treated patients in EMBARK, the company’s global, randomized placebo-controlled Phase 3 study evaluating ELEVIDYS in ambulatory individuals living with #Duchenne.

Recently @Solid_Bio shared updates on the company’s gene therapy program for the treatment of #Duchenne, with 33 participants now dosed in the ongoing Phase 1/2 INSPIRE DUCHENNE clinical trial of SGT-003. parentprojectmd.org/solid-bioscien…

Registration is open for PPMD’s 2026 Advocacy Conference, taking place march 8-10 in Washington, DC! Learn more and register today: donate.parentprojectmd.org/event/ppmd-adv… #PPMDAdvocacy

As we embark on 2026, there’s still time to make a gift to PPMD to help us #HonorEveryJourney of every person living with #Duchenne and #Becker. From now through midnight 12/31, every gift will be DOUBLED. Let’s make this year one of hope—for ALL. donate.parentprojectmd.org/give/733746/#!…

Help PPMD #HonorEveryJourney this holiday season. Give today and your donation will go twice as far, thanks to a match provided by John Owen’s Adventure, Gail & David Hofstein, the Alex Rezkalla Family, and other community members. donate.parentprojectmd.org/give/733746/#!…