Muscular Dystrophy Association

We've resubmitted our BLA to the FDA for our investigational, muscle-targeted treatment for SMA! Our progress reaffirms the promise of myostatin biology as we continue to prioritize scientific advancement & the needs of children & adults living with SMA. bit.ly/4sIxX9x

📢 Big news for the SMA community: The FDA has approved a high-dose SPINRAZA regimen for SMA—another major step forward in care. Developed by @biogen, this milestone builds on decades of MDA-supported research. Progress keeps moving 🔗 mda.org/press-releases… #SMA #Research

MDA is grateful to our Strength of Life Sponsors: @argenxglobal, @biogen, @BridgeBioPharma, @Novartis for their continued commitment to bringing the #neuromuscular community together at the 2026 #MDAconference. ➡️Register today: mdaconference.org

Register now! mdausa.webex.com/weblink/regist…

Family caregivers are essential to the neuromuscular community. Join MDA Advocacy on 3/2 at 12 PM ET for a special MDA Advocacy Institute spotlight on the Credit for Caring Act & Alleviating Barriers for Caregivers Act. Hear from Capitol Hill + AARP & learn how to take action.

3/3 ➡️ Next stop: the U.S. House for final passage. 📣 Take action—contact your Representative and urge them to support this bill: 🔗 votervoice.net/MDA/Campaigns/…

2/3 The bill also supports kids & families by including the Accelerating Kids’ Access to Care Act and reauthorizing the Rare Pediatric Disease Priority Review Voucher program. ✅

1/3 🚨 BREAKING: The U.S. Senate just passed legislation advancing major priorities for the neuromuscular disease community—including strong NIH & federal medical research funding.

What’s on the public policy horizon for 2026? 🏛️ Join us for the #MDA #Advocacy Institute on Jan. 29 at 7 PM ET to learn about our 2026 advocacy agenda & how the #neuromuscular community can add their voices. Register: mdausa.webex.com/weblink/regist…

🚨 NIH funding vote THIS WEEK. Rare disease research is on the line. 🦓 We dropped a breaking news episode of DNA Today (Ep. 378) on what’s at stake. 👉 Senate vote this week so urger your senators to support the NIH with @MDAorg's tool: MDA.org/SupportNIH

Caregivers face growing challenges as support systems fall behind. MDA is advocating for solutions, and we need your input. 🏛️✨ 🗣️ Share your caregiving experiences: votervoice.net/MDA/Surveys/13… #Caregiving #MDA #Advocacy #DisabilityPolicy

(3/3) MDA extends its appreciation to the premier sponsors, including: Strength of Life: @argenxglobal, @Biogen, @BridgeBioPharma, @Novartis Circle of Strength: ITF Therapeutics, @REGENXBIO, @ScholarRock #MDAconference #Research #Leadership #MDA

(2/3) These leaders will be celebrated at the MDA Clinical and Scientific Conference in Orlando this March. 🧬👏 ➡️ Read the full press release to learn more about their groundbreaking work and lasting legacy: mda.org/press-releases… ➡️ Register Today: mdaconference.org

(1/3) 🎉 The Muscular Dystrophy Association announces the 2026 MDA Legacy Award recipients: Dr. Michio Hirano (Columbia University Irving Medical Center) for lifetime clinical research achievement, and Allison Moore (HNF) for transformative community impact.

Yesterday, Solid Biosciences shared an important update with the Friedreich’s ataxia community. The first participant has been dosed in the Phase 1b FALCON trial evaluating SGT-212, our novel, investigational dual-route administration gene therapy for the treatment of FA. We are deeply grateful to the FA community, @CureFA_org, our clinical partners, and the patients and families whose trust and participation make this work possible. Your partnership continues to guide our mission to advance meaningful therapies for people living with FA. Read the full community letter here: solidbio.com/letter-to-the-… #FriedreichsAtaxia #FACommunity #RareDiseaseResearch

The Muscular Dystrophy Association has opened applications for its 2026 college scholarship program for students with neuromuscular disease. musculardystrophynews.com/news/applicati… #musculardystrophynews #musculardystrophy #musculardystrophycommunity #livingwithMD

You may know me from Grey’s Anatomy as Joe the bartender — or from appearances on Modern Family, You, Chicago Fire, or some other appearance. I would like to share something important with you about my life and my career. 🧵👇

Amyotrophic lateral sclerosis is a progressive neurodegenerative disease. Here's what you need to know about ALS, from risk factors to new treatments, medications, and clinical trials. health.usnews.com/conditions/bra…

A historic year. Real progress. A community moving #ForwardTogether 💙💛 In her first End of Year message, MDA President & CEO Sharon Hesterlee, PhD reflects on MDA’s 75th anniversary and how 2025 set the stage for what’s next. Read more: mdaquest.org/a-year-built-t… #MDA75

Cutting NIH funding stalls life-changing progress. As Congress debates NIH and medical research, remind them who they fight for—families like Katie’s. Take action with MDA Ambassador Katie Brooks. Join us: #SupportNIH 👉 mda.org/supportNIH