Together, we have made remarkable progress in #Duchenne and #Becker care and research, and 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.
With your help, we’re identifying gaps in care, analyzing clinic practices, and developing a roadmap for high-quality adult care, ensuring adults with Duchenne and Becker have access to the coordinated, compassionate support they deserve.
❤️ Make a donation today to support PPMD’s Adult Care Initiative. Thanks to the Nicholoff Family, every gift will be DOUBLED up to $75,000.
Give now at the link in our bio.
📢 News: Today, Gemma Biotherapeutics (GEMMABio), a clinical‑stage, global, genetic therapeutics company, shared information about the company’s #Duchenne #musculardystrophy program, presenting preclinical data for GB703, a novel, investigational #genetherapy for the treatment of Duchenne.
PPMD is excited to see a new potential gene therapy candidate in early development that may offer solutions to current challenges and we welcome GEMMABio to the Duchenne community.
Learn more at the link in our bio.
📢 News: REGENXBIO Inc. has announced positive topline and interim functional data from the pivotal Phase III portion of the Phase I/II/III AFFINITY DUCHENNE® trial of RGX-202, an investigational #genetherapy being developed for individuals with #Duchenne.
In addition to favorable safety and biomarker data, the trial met its primary endpoint of at least 10% microdystrophin expression at week 12, with microdystrophin expression also demonstrating statistically significant correlation with functional improvement at one year.
“For decades, our community has pushed for therapies that can change the trajectory of this disease, and today’s news gives us renewed optimism,” said PPMD’s Pat Furlong.
Learn more at the link in our bio.
❗Don’t miss your chance to save on registration for PPMD’s 2026 Annual Conference — early bird pricing ends TOMORROW!
Join the #Duchenne and #Becker community for a powerful 3 days of connection, education, advocacy, and hope. From research and care updates to community support, networking, and tons of fun, the Annual Conference brings families, clinicians, researchers, industry partners, and advocates together like nowhere else.
We can’t wait to see you in Orlando, June 25-27! Register today at the link in our bio.
❤️ Happy Mother’s Day from PPMD! ❤️
Whether they are celebrating milestones or holding families together as we navigate this journey, these remarkable women are the heart of our community. Their courage inspires hope, their advocacy drives progress, and their love changes lives.
Honor the moms, grandmas, and mother figures in your life with a gift to PPMD and send her an eCard to show your love and appreciation for all she does. Visit the link in our bio.
❤️ Mother’s Day is around the corner.
This year, honor the extraordinary moms, grandmas, and mother figures in the Duchenne and Becker community whose love, strength, and unwavering devotion make a lasting difference in your life every day, by making a donation to PPMD.
Send an eCard and make a gift to celebrate her legacy while helping move us closer to better treatments, stronger support, and brighter tomorrows for everyone affected by Duchenne and Becker.
Give for Mother’s Day at the link in our bio.
Across the country, we are seeing strong interest from states as they explore adding #Duchenne to their #newbornscreening programs.
We’re excited about recent updates out of Tennessee and Nebraska; this June marks the deadline for Massachusetts to begin screening; and we expect Arizona and other RUSP-aligned states to start in 2027.
This progress is a direct result of families, clinicians, researchers, and partners across the Duchenne community raising their voice. Learn more about newborn screening progress and how you can get involved at the link in our bio.
You can now check out the PPMD 2026 Annual Conference Agenda at a Glance for a preview of what you can expect—from in-depth educational sessions and expert-led panels, to interactive workshops and tailored specialty tracks designed to help you navigate every aspect of the #Duchenne and #Becker journey.
Whether you’re attending for the science, the support, or the sense of community, there’s something at Annual Conference for everyone!
Early bird registration for PPMD’s 2026 Annual Conference ends MAY 14. Register today and get ready to join us in Orlando next month!
PPMD, in collaboration with the Muscular Dystrophy Association (MDA) and the Little Hercules Foundation (LHF), convened a Duchenne Newborn Screening (NBS) Stakeholder Meeting last week to gather industry partners in #Duchenne to establish a foundation of understanding of the process of state implementation, and to commit to a shared vision and resource sharing across all partners.
We’re proud to be able to bring together leaders across advocacy, public health, and policy to align on the current landscape and accelerate a shared vision for implementing Duchenne NBS across the U.S.
Learn more about our NBS advocacy efforts at the link in our bio.
Join PPMD on Thursday, April 30th at 1:00 PM ET for a webinar, “Exercise and Dystrophinopathy: What Families Need to Know,” designed to help families better understand how exercise research is being translated into real-world care and share resources for practical, family-centered insights for daily life and long-term health.
Register today and submit questions in advance at the link in our bio.
From meaningful connections to unforgettable Disney moments, the 2026 PPMD Annual Conference is packed with special events designed to bring the PPMD community together.
Kick off this year’s Conference with a Newly Diagnosed Meet & Greet and an evening Teen & Adult Social hosted by PPMD’s Adult Advisory Committee (PAAC). And don’t miss our Welcome Reception & Resource Fair, featuring 40+ vendors, great food, and quality community time. We’ve even penciled in a flexible early wrap-up on Friday, perfect for heading to the parks with family and friends!
Be sure to stick around on Saturday for an exclusive evening at Disney’s Animal Kingdom®, featuring a hosted dinner and a private performance of Festival of the Lion King!
Come for the information, stay for the connection—and experience a little magic along the way. Register for PPMD’s 2026 Annual Conference by May 14 for early bird rates!
PPMD has a long history of working alongside our community to document treatment preferences and share those insights with regulatory authorities such as the FDA. Through town halls, listening sessions, and conversations at community events, we consistently hear that stability is a critically important outcome in both care and treatment.
PPMD has collaborated with researchers at The Ohio State University to conduct a patient preference survey that aims to better understand how individuals with #Duchenne and their caregivers value stability as an outcome of therapeutic intervention, and to capture what stability truly means in the context of treatment and care.
BOTH caregivers and individuals living with Duchenne from the same household are welcome to take the survey! Each person who wants to participate must complete the interest form in order to receive their own individualized survey link.
Fill out the interest form at the link in our bio.
