Parent Project Muscular Dystrophy (PPMD)

#EndDuchenne

[Parent Project Muscular Dystrophy (PPMD) @ParentProjectMD]

With your help, we’re identifying gaps in care, analyzing clinic practices, and developing a roadmap for quality care, ensuring adults have access to the coordinated, compassionate support they deserve. Give today to support PPMD’s Adult Care Initiative: https://t.co/xEGZPMZNAx

[Parent Project Muscular Dystrophy (PPMD) @ParentProjectMD]

While adulthood is now possible for many in our community, adequately coordinated care systems have not kept pace.

⭐ PPMD’s Adult Care Initiative aims to establish the gold standard of care for adults living with #Duchenne and #Becker.

[Parent Project Muscular Dystrophy (PPMD) @ParentProjectMD]

Navigating #Duchenne #musculardystrophy genetics can feel overwhelming, but you don’t have to do it alone. We’re joining @JettFoundation Foundation for a webinar on 4/30 to help families better understand the genetic side of Duchenne. Register now: https://t.co/y5GzuL9JOX

[Parent Project Muscular Dystrophy (PPMD) @ParentProjectMD]

PPMD has provided $250,000 in funding to @SecretomeTx through PPMD Venture Pathways to advance the development of STM-01, Secretome's neonatal cardiac progenitor cell (nCPC) product for the treatment of #Duchenne -associated cardiomyopathy. Learn more:
https://t.co/cedfBEMCps

[Parent Project Muscular Dystrophy (PPMD) @ParentProjectMD]

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: https://t.co/nTtn9oD0W7

[Parent Project Muscular Dystrophy (PPMD) @ParentProjectMD]

��� 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: https://t.co/bQrTi0OpVz

[Parent Project Muscular Dystrophy (PPMD) @ParentProjectMD]

📢 News: @Solid_Bio has shared updated positive interim data from the ongoing Phase 1/2 INSPIRE DUCHENNE clinical trial of SGT-003. Learn more: https://t.co/01kUvX2pt3

[Parent Project Muscular Dystrophy (PPMD) @ParentProjectMD]

📢 News: @REGENXBIO has announced new positive interim data from the Phase I/II AFFINITY DUCHENNE trial of RGX-202. Learn more: https://t.co/JXub7Ur3S5

[Parent Project Muscular Dystrophy (PPMD) @ParentProjectMD]

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

https://t.co/4KSOksyKn9

[Parent Project Muscular Dystrophy (PPMD) @ParentProjectMD]

🏛️ 📣 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

[Parent Project Muscular Dystrophy (PPMD) @ParentProjectMD]

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: https://t.co/ky4i8e5n9T

[Parent Project Muscular Dystrophy (PPMD) @ParentProjectMD]

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.

[Parent Project Muscular Dystrophy (PPMD) @ParentProjectMD]

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.

[Parent Project Muscular Dystrophy (PPMD) @ParentProjectMD]

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:
https://t.co/yTBuMxZL81

[Parent Project Muscular Dystrophy (PPMD) @ParentProjectMD]

@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: https://t.co/1KdzJaf3c5

[Parent Project Muscular Dystrophy (PPMD) @ParentProjectMD]

@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: https://t.co/TofimioIy1

[Parent Project Muscular Dystrophy (PPMD) @ParentProjectMD]

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!

[Parent Project Muscular Dystrophy (PPMD) @ParentProjectMD]

✨ 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. https://t.co/sg4H5ru7bT

[Parent Project Muscular Dystrophy (PPMD) @ParentProjectMD]

❗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. https://t.co/zRSBW7M3Mo

[Parent Project Muscular Dystrophy (PPMD) @ParentProjectMD]

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.