Muscular Dystrophy Association Clinical and Scientific Conference (MDA)
The Muscular Dystrophy Association (MDA) Clinical and Scientific Conference highlights unprecedented research advancements and clinical achievements in neuromuscular disorders and includes patients, physicians, researchers, and more, with topics of discussion focusing on all aspects of preclinical, translational, and clinical research and care.
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.
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]
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]
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]
Clinical Application of Induced Pluripotent Stem Cells in Muscular Dystrophy: Rita Perlingeiro, PhD
April 13th 2023The Lillehei professor in stem cell and regenerative cardiovascular medicine at the University of Minnesota talked about the potential of investigating inducted pluripotent stem cells in neuromuscular diseases at MDA’s 2023 conference. [WATCH TIME: 5 minutes]
Assessing New Long-Term Efficacy and Durability Data of Zolgensma for Spinal Muscular Atrophy
April 11th 2023Sitra Tauscher-Wisniewski, MD, vice president clinical development and analytics, Novartis Gene Therapies, talked about the new data findings from a study on gene therapy Zolgensma at the 2023 MDA conference.
Predicting Clinical Needs and Potential for Genetic Therapies in ALS: Jennifer Morganroth, MD, MBA
April 11th 2023At the 2023 MDA’s Clinical and Scientific conference, the neurology resident at Penn Medicine talked about the challenges of delivering intrathecal therapies and the importance of planning clinical resources for patients with ALS. [WATCH TIME: 3 minutes]
Revolutionizing Treatment For Duchenne Muscular Dystrophy with Gene Editing: Courtney Young, PhD
April 10th 2023The co-founder and chief executive officer of MyoGene Bio talked about the advantages of gene editing over traditional gene therapy as a long term treatment for neuromuscular diseases at MDA’s 2023 conference. [WATCH TIME: 3 minutes]
Physical Therapy, Optimizing Function in Neuromuscular Diseases: Megan Iammarino, PT, DPT
April 9th 2023The research physical therapist at Nationwide Children’s Hospital talked about the role of physical therapy in the changing landscape of care with neuromuscular diseases at the 2023 MDA conference. [WATCH TIME: 5 minutes]
The Development of New Gene Therapies to Address Unmet Needs in ALS: Matthew B. Harms, MD
April 7th 2023The associate professor of neurology at Columbia University spoke at the 2023 MDA conference about the unmet needs of patients with ALS and the potential of new gene therapies. [WATCH TIME: 5 minutes]
Early Monitoring and Treatment for Bone Health in Duchenne Muscular Dystrophy: Meilan Rutter, MD
April 6th 2023At the 2023 MDA conference, a pediatric endocrinologist from Cincinnati Children’s Hospital talked about the unmet needs in treating bone health for patients with Duchenne muscular dystrophy. [WATCH TIME: 5 minutes]
Personalized Treatment Approaches of Myopathies Based on Antibodies: Suur Bilicier, MD
April 5th 2023The associate professor of neurology at the University of Texas Health Science Center at Houston talked about advances in the classification of inflammatory myopathies at the recently concluded 2023 MDA conference. [WATCH TIME: 4 minutes]
Top Interviews from 2023 MDA: Expert Insights on Neuromuscular Diseases
April 4th 2023A group of experts in the care of patients with neuromuscular diseases—Douglas Sproule, MD, MSc; David Brumbaugh, MD; Sharon Hesterlee, PhD; Lauren Treat, MD; Donovan Lott, PT, PhD, CSCS—shared their perspectives on hot topics of treatment and management from the 2023 MDA conference.
The Potential of Genetic Therapies in Neuromuscular Diseases: Stephan Züchner, MD, PhD
April 4th 2023At the 2023 MDA conference, the professor for human genetics and neurology at the University of Miami Miller School of Medicine talked about the role of genetics in neuromuscular diseases and potential therapies. [WATCH TIME: 5 minutes]
Lessons Learned and Future Possibilities in ALS Collaborations: Edritz Javelosa, PhD
April 4th 2023At the 2023 MDA conference, the scientific research portfolio director at Muscular Dystrophy Association detailed a session on collaborative research in ALS and ongoing efforts in the field. [WATCH TIME: 3 minutes]
Focus on Gene Therapy and Neuroinflammation in ALS Research: Stanley H. Appel, MD
April 3rd 2023The director of the Ann Kimball and John W. Johnson Center for Cellular Therapeutics at Houston Methodist Hospital spoke on about advances in ALS research and promising therapies on the horizon at the 2023 MDA conference. [WATCH TIME: 8 minutes]
Holistic Approach to Medicine and Rehabilitation in Physical Therapy: Tina Duong, PhD, PT
April 2nd 2023The director of clinical outcomes and research development in neurology at Stanford University spoke about the paradigm shift in neuromuscular therapy interventions at the 2023 MDA conference. [WATCH TIME: 5 minutes]
Advances in Gene Therapy for Neuromuscular Diseases: Kevin Flanigan, MD
March 31st 2023The director of the center for gene therapy at Nationwide Children's Hospital talked about the challenges and opportunities of gene therapies for neuromuscular diseases at the 2023 MDA conference. [WATCH TIME: 4 minutes]
