Washington, D.C.
Joined June 2009
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What better moment to introduce the #SurgetoSaveNewborns Act than #NewbornScreeningAwarenessMonth? Now is the time to urge Congress to pass this critical legislation: surgetosave.org/act-now/
Today, @Capricor announced that the FDA has accepted the company’s amendment to the BLA for Deramiocel, and the FDA will be extending the current PDUFA action date to November 22, 2026.
Learn more and catch up on recent Deramiocel updates: parentprojectmd.org/update-o…
As we approach FDA’s current 8/22 PDUFA date for Deramiocel, the #Duchenne community is watching closely for an update. We've requested an update from @Capricor and will share information with the community as soon as possible. parentprojectmd.org/deramioc…
The next PPMD Livestream with the PAAC is coming Thursday 8/27 at 4 PM ET! Join us for real-life tech tips, tools, and hacks to make life more accessible, manageable, and independent for people living with #Duchenne or #Becker. Tune in next week! facebook.com/share/1L3qzFffr…
PPMD, in collaboration with #Duchenne advocacy partners Best Day Ever Foundation, @CureDuchenne, @JettFoundation, @KindnessOverMD, @LHF_EndDuchenne, @MDAorg, MDFF, and @TeamJosephInfo, invites you to join a webinar on July 7 at 2 PM ET...
Register now: parentprojectmd.org/events/w…
The webinar will focus on how our community can engage in the upcoming FDA Advisory Committee meeting for Deramiocel on July 29. Join us to learn how you can be part of this important moment for the Duchenne community. Register now: parentprojectmd.org/events/w…
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: donate.parentprojectmd.org/c…
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: jettfoundation.org/event/com…
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:
parentprojectmd.org/ppmd-pro…
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/e…
📢 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…
📢 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-bi…
📢 News: @REGENXBIO has announced new positive interim data from the Phase I/II AFFINITY DUCHENNE trial of RGX-202. Learn more: parentprojectmd.org/regenxbi…
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…
🏛️ 📣 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
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.
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.
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-par…
