Muscular Dystrophy Association

🎉 Congratulations to Mike Bush on 40 incredible years with KSDK - 5 On Your Side News! Your dedication, storytelling, and heart have made a lasting impact on countless lives. We’re looking forward to honoring this incredible milestone with you and the neuromuscular disease community at the Show of Strength event in St. Louis on August 31 in support of MDA families across the country. 💙 💛

📣 Registration is now OPEN! Join us at the 2026 MDA Clinical & Scientific Conference — the largest global gathering of the neuromuscular disease community — happening March 8-11 in Orlando, Florida. Connect with world-renowned experts advancing scientific research and clinical care. 🔗 Register now at https://xmrrwallet.com/cmx.plnkd.in/g9NtQfY Submit your abstract for oral presentation consideration here: https://xmrrwallet.com/cmx.plnkd.in/gWkYyB9Q Read more in the press release here: https://xmrrwallet.com/cmx.plnkd.in/gzNgtzKk #MDAconference #Neuromuscular #RareDisease #GeneTherapy #ClinicalTrials

In this powerful Quest podcast episode, host Mindy Henderson sits down with current and former MDA National Ambassadors Payton Rule, Fred Graves, and Amy Shinneman to talk about transformation, resilience, and what Disability Pride means to each of them. 💬 Payton opens up about finding pride through community. 💪 Fred shares how focusing on his strengths helps him rise above daily challenges. 💖 Amy reflects on embracing her disability as a core part of her identity. Together, they explore the beauty of authenticity, the importance of representation, and how embracing who you are can lead to real empowerment. ✨ 🎧 Listen to the full episode at https://xmrrwallet.com/cmx.plnkd.in/gABYGcst #DisabilityPride #QuestPodcast #MDA #MuscularDystrophy #MDAambassadors #DisabilityAwareness #Empowerment #DisabilityAdvocate

CureLGMD2i is hosting an Advocacy Power Hour on Tuesday, August 12th at noon EDT. Join us for a powerful one-hour session, featuring Paul Melmeyer (Muscular Dystrophy Association) & Karin Hoelzer (Biotechnology Innovation Organization), and hosted by Kelly Brazzo & Dan Pope (CureLGMD2i Foundation) as we share the latest advocacy updates, policy priorities, and rare disease community-driven efforts shaping the future for those living with rare conditions like the LGMDs. Topics will cover recent milestones such as the pursuit and impact of standards of care and ICD-10 codes, the growing collaboration among our coalition of LGMD patient advocacy orgs, and patient-led efforts to influence legislation, research funding, and FDA engagement for the future of approved therapies for rare diseases. Register here: https://xmrrwallet.com/cmx.plnkd.in/eEbiANdZ This program is sponsored by a grant from the Muscular Dystrophy Association.

We are deeply saddened by the passing of Phil Green, an advocate, devoted husband and father, and a powerful voice in the ALS community. Phil lived with purpose and passion, dedicating each day to advancing access to treatments, driving research for biomarkers, and championing legislation to improve the lives of people living with ALS. Phil once said, "I get up each day motivated to make a difference... I bring the urgency and passion of the patient voice." His legacy will continue to inspire us in the fight to end ALS. MDA extends our heartfelt condolences to Phil’s family, friends, and the entire ALS community. 💙💛 #ALS #EndALS

The Muscular Dystrophy Association has been notified of Sarepta Therapeutics’ announcement yesterday that it has temporarily paused all U.S. shipments of ELEVIDYS, including for ambulatory patients. According to Sarepta, this pause is intended to allow for discussions with the U.S. Food and Drug Administration (FDA) regarding potential updates to the treatment’s label. We know how significant and personal this news is for families who were preparing to receive treatment. We also understand the uncertainty this may create for families navigating the complex landscape of gene therapy options for Duchenne muscular dystrophy (DMD). MDA remains dedicated to our belief in the transformative potential of gene therapy to change the course of neuromuscular diseases. At the same time, we recognize that access to any therapy must be guided by rigorous scientific evidence, regulatory oversight, and a consistent and abiding commitment to patient safety. We urge families impacted by this development to speak directly with their MDA Care Center team. Families may also reach out to the MDA Resource Center for further assistance at 1-833-ASK-MDA1 (1-833-275-6321) or ResourceCenter@mdausa.org. #DuchenneMuscularDystrophy #MDA #GeneTherapy #MuscularDystrophy #NeuromuscularDisease

July is Disability Pride Month 💪 You don’t have to apologize for who you are. Your disability is not something to hide. It’s a part of what makes you strong, unique, and powerful! 💙💛 Celebrate Disability Pride Month and subscribe to Quest Media for FREE! https://xmrrwallet.com/cmx.plnkd.in/eFNHRRAj #DisabilityPrideMonth #DisabledAndProud #MDA

🔔 Save the Date Life Takes Muscle: Perspectives on the Remaining Unmet Needs in SMA Treatment 📅 Wednesday, July 30 | 2:00 PM ET 🕒 Duration: 30 minutes 📍 Live Webinar | Presented by Scholar Rock Join MDA for an educational session tailored to all healthcare providers working with individuals living with spinal muscular atrophy (SMA). This 30-minute webinar will explore: ✅ The unmet needs in SMA treatment, particularly from a muscle-focused approach ✅ The vital role of muscle in everyday life ✅ Personal perspectives from people living with SMA on their treatment journey and aspirations for improved motor function Don't miss this opportunity to deepen your understanding of SMA care and support better outcomes for patients. 🔗 Register here: https://xmrrwallet.com/cmx.plnkd.in/gNEHDGNE #SMA #NeuromuscularCare #SMAawareness #MDA #NeuroMuscular #MuscularDystrophy

Read the statement at Sarepta.com.

We are heartbroken by the recent loss of an MDA community member in a gene therapy trial for limb-girdle muscular dystrophy (LGMD). Our deepest condolences go out to loved ones and to all in the LGMD community who are grieving. We also recognize concerns in the Duchenne muscular dystrophy (DMD) community following news that the FDA has asked to pause shipments of Elevidys after two adolescent patients died earlier this year. These developments are painful, and they’ve shaken the hope that many families have carried for years. Gene therapy remains one of the most hopeful frontiers in neuromuscular research. These painful losses remind us how complex and fragile progress can be and how essential it is that safety remains at the center of every step forward. We’re encouraged to see the FDA working closely with researchers, companies, and patient advocates to strengthen safeguards and ensure responsible momentum across all emerging treatments. MDA will continue to work with the pharmaceutical industry and FDA for safe, effective options. MDA’s mission remains as clear as ever: we believe in the future of gene therapy, but our commitment to safety is non-negotiable. We are here to support every person and family affected by LGMD and DMD as we continue pushing forward together. Families may contact the MDA Resource Center for guidance and support at 1-833-ASK-MDA1or email ResourceCenter@mdausa.org.