Muscular Dystrophy Association

From 25+ FDA-approved therapies to expert care for 70,000+ people each year, IAFF members are creating real change with @MDAorg. Read more about the @IAFFofficial's Fill the Boot impact in the latest issue of our Fire Fighter International magazine:

Read the statement at Sarepta.com.

MDA Statement to our Community: mda.org/press-releases…

Students with neuromuscular diseases deserve schools that support their needs. [TAG] @MDA_Advocacy works to strengthen IEPs, improve access & fight for inclusive policies. ➡️ Learn more: mdaquest.org/understanding-… #MDAadvocacy #DisabilityRights #EducationPolicy

We were honored to attend the @MDAorg's 25th Annual Wings Over Wall Street Gala in NYC! We joined researchers, advocates & families, all united by one cause – a world free from ALS. 25 years strong and still going. #WOWS25 #ALSAdvocacy

Families affected by #Duchenne #MuscularDystrophy (DMD) may be eligible for @REGENXBIO’s Phase 3 trial of RGX-202, an investigational gene therapy delivering a functional dystrophin gene. Read the full study alert: mdaquest.org/clinical-resea… #GeneTherapy #ClinicalTrials #MDA75

Congress has passed legislation that makes historic cuts to #Medicaid, #Medicare, and #ACA, & it could leave 17M more uninsured, including those w/ #neuromuscular diseases.   #MDA is disappointed, and we pledge to defend our community.

Today, the U.S. Senate passed a bill making devastating cuts to Medicaid & other health programs that the #neuromuscular community relies on to build healthy lives & independence. Urge the House to reject this bill & its cuts NOW! Read #MDA's statement: mda.org/press-releases…

🚨 NEW: MDA + @CureFA_org are co-funding a $300K research grant to study cardiac fibrosis in Friedreich’s ataxia (FA), a key cause of heart failure in FA. This study could lead to life-changing therapies. 🔗 bit.ly/46nuqoB #FriedreichsAtaxia #CardiacResearch

Today, @MDAorg & 41 #NMD orgs. sent a letter to U.S. Senate leadership rejecting cuts to #Medicaid & affordable health insurance coverage/services used by the #neuromuscular community - raising costs, imposing red tape, & delaying care. Read the letter: d3dkdvqff0zqx.cloudfront.net/groups/mda/att…

Today we announced new positive interim functional data from our Phase I/II AFFINITY DUCHENNE trial of RGX-202. Learn more: ir.regenxbio.com/news-releases/… $RGNX

The Administration for Community Living (ACL) is facing serious threats. Learn why defending the ACL matters, what’s at stake, and how you can be part of protecting these essential programs. 👉 Read the full blog: mdaquest.org/understanding-…

Today, we announced that the FDA has granted Fast Track designation to our investigational ASO therapy for the treatment of ALS, which is currently being studied in the Phase 1 LUMINA trial. Early cohort data from LUMINA is expected in 2025. We are committed to advancing this…

This MDA Ambassador was representing last year at Angels Stadium with the @MDAorg and @Angels for Lou Gerhig Day! I am Smashing Pompe! Swing Away 4 Pompe!