Muscular Dystrophy Association | Strategic Alliance Partners
Muscular Dystrophy Association (MDA) is the #1 voluntary health organization in the United States for people living with muscular dystrophy, ALS, and related neuromuscular diseases. For over 70 years, MDA has led the way in accelerating research, advancing care, and advocating for the support of our families. MDA's mission is to empower the people we serve to live longer, more independent lives.
Latest from Muscular Dystrophy Association
A Patient Perspective on Gene Therapy for Neuromuscular Diseases: Justin Moy
March 26th 2024The second-year PhD student in bioinformatics at Boston University who lives with LAMA2 congenital muscular dystrophy talked about the potential impact and challenges of gene therapy in neuromuscular diseases. [WATCH TIME: 5 minutes]
Intravenous Zolgensma Shows Motor Function Improvement in Heavier Pediatric Patients With SMA
March 7th 2024Findings from the recent phase 3b SMART trial affirm the safety and efficacy of intravenous onasemnogene abeparvovec (Zolgensma; Novartis) in spinal muscular atrophy when patient weights range from 8.5 kg to 21 kg.
Respiratory Patterns Differ in Pediatric Duchenne Muscular Dystrophy
March 6th 2024A recent study presented at MDA 2024 highlighted the evolving respiratory patterns in pediatric patients with Duchenne muscular dystrophy, offering crucial insights for effective respiratory management in this patient population.
Preoperative Disease-Modifying Agents Reduce Postoperative Complications in SMA Scoliosis Surgery
March 5th 2024A recent study presented at MDA 2024 suggests that preoperative use of disease-modifying agents for patients with spinal muscular atrophy leads to less severe postoperative complications following scoliosis surgery.
Viltolarsen Shows Significant Pulmonary Function Gains in Ambulant and Nonambulant DMD
March 5th 2024In a recent phase 2 trial analysis of viltolarsen presented at MDA 2024, findings showed improvement in forced vital capacity compared with standard care in patients with Duchenne muscular dystrophy.
Meta Analysis Shows Slow Decline of Muscle Function With Ataluren for Nonsense Mutation DMD
March 5th 2024A new meta analysis of 3 studies presented at MDA 2024 revealed that ataluren significantly slowed the decline in muscle function for patients with nonsense mutation Duchenne muscular dystrophy.
Post Hoc Analysis Shows Consistent Efficacy With Givinostat for DMD
March 5th 2024A recent post hoc analysis of the phase 3 EPIDYS trial presented at MDA 2024 revealed significant positive outcomes with givinostat, a histone deacetylase inhibitor, among patients with Duchenne muscular dystrophy.
Reaction to 2024 MDA Legacy Award: Jeff Chamberlain, PhD
March 3rd 2024Jeff Chamberlain, PhD, a leading professor in gene therapy focused on Duchenne muscular dystrophy, shared his reaction to being named the recipient of the 2024 MDA Legacy Award, as well as the state of the DMD field currently.
Challenges for the Neuromuscular Community Entering Treatment Era: Barry Byrne, MD, PhD
March 1st 2024The chief medical advisor at the Muscular Dystrophy Association provided perspective on the upcoming meeting and the conversations surrounding new therapeutics for diseases that once had little to nothing. [WATCH TIME: 4 minutes]
Advocacy and Progress in Rare Neuromuscular Diseases From a Policy Standpoint: Paul Melmeyer, MPP
February 29th 2024In honor of Rare Disease Day, the vice president of public policy and advocacy at the Muscular Dystrophy Association shared his perspective of advocacy and policy progress in the realm of rare neuromuscular diseases. [WATCH TIME: 6 minutes]
Future Role of Neurofilament Light in ALS Drug Development, Clinical Trials: Rita Sattler, PhD, MSc
February 23rd 2024The professor in the Department of Translational Neuroscience at Barrow Neurological Institute provided perspective on the approval of tofersen (Qalsody; Biogen) for patients with ALS and the ways neurofilament light will be used going forward. [WATCH TIME: 3 minutes]
Highlighting Genetic Discoveries and Therapeutic Developments in ALS: Matthew B. Harms, MD
February 21st 2024The associate professor of neurology at Columbia University talked about a track session that he will be chairing at the upcoming MDA conference on amyotrophic lateral sclerosis genetic research. [WATCH TIME: 5 minutes]
NeuroVoices: Rita Sattler, MD, MSc, on 2024 MDA Conference and Advances in ALS Research
February 21st 2024The professor in the Department of Translational Neuroscience at Barrow Neurological Institute discussed several topics related to ALS research and the emerging biomarkers in recent years ahead of the 2024 MDA Clinical and Scientific Conference.
The Emergence of Novel Biomarkers and Model Systems for ALS: Rita Sattler, MD, MSc
February 19th 2024The professor in the Department of Translational Neuroscience at Barrow Neurological Institute provided comments on her session track at the upcoming 2024 MDA conference and some of the recent advancements in ALS research. [WATCH TIME: 4 minutes]
Advancements and Challenges in Genetic Treatments for Neuromuscular Diseases: John W. Day, MD, PhD
February 12th 2024Ahead of the 2024 MDA Conference, the medical advisor and care center director at Muscular Dystrophy Association talked about the track sessions he will be cochairing at the meeting. [WATCH TIME: 5 minutes]
How Clinicians can Advocate for Patients Beyond the Clinic: Michael Lewis, MA
November 7th 2023The director of disability policy at the Muscular Dystrophy Association discussed his lecture at AANEM 2023 on how clinicians can advocate for their patients to help improve accessibility and health equity. [WATCH TIME: 7 minutes]
Clinical Viewpoint on Newly Approved Combination Therapy for Pompe Disease: Barry J. Byrne, MD, PhD
October 5th 2023The chief medical advisor at the Muscular Dystrophy Association gave his reaction to Amicus Pharmaceuticals’ recently FDA approved 2-part therapy for patients living with Pompe disease. [WATCH TIME: 7 minutes]
