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Muscular Dystrophy Association Clinical and Scientific Conference

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.

Gaining a Clearer Picture of Gene Therapy in Neuromuscular Disease: Carsten G. Bönnemann, MD

March 23, 2022

The senior investigator in the Neuromuscular and Neurogenetic Disorders of Childhood Section at NINDS shared his perspective on the data coming out of neuromuscular research and the promise of genetic medicine. [WATCH TIME: 2 minutes]

Jinsy Andrews, MD, MSc, FAAN, on Engaging With Patients and Colleagues Amid an Advancing Neuromuscular Field

March 22, 2022

The director of neuromuscular clinical trials at Columbia University Irving Medical Center shared her perspective on the discussions that took place at MDA 2022 and the importance of collaboration among clinicians and researchers.

The Need for Participation in Neuromuscular Disease Clinical Trials: Donald S. Wood, PhD

March 22, 2022

The president and CEO of the Muscular Dystrophy Association spoke about the need for increased participation in neuromuscular disorder clinical trials and the rapid therapeutic progress the field is seeing. [WATCH TIME: 4 minutes]

BBP-418 Demonstrates Significant Impact on Root Cause of Limb-Girdle Muscular Dystrophy Type 2i

March 22, 2022

Eleven of the 12 individuals demonstrated at least a 50% reduction in creatine kinase, with 75% of participants reaching twice the normal range, suggesting a reduction in muscle breakdown.

Gene Therapy Zolgensma Improves Bulbar Function in Symptomatic SMA Type 1

March 21, 2022

More than 90% of the 65-patient cohort had evidence of normal swallowing and had no aspiration or pneumonia aspiration events reported in the phase 1 START and phase 3 STRIVE-EU and STRIVE-US trials.

Successes and Challenges in the New Era of Genetic Medicine: Nicholas E. Johnson, MD, MSci

March 20, 2022

The division chief of neuromuscular disorders and vice-chair of research at Virginia Commonwealth University spoke to the ongoing success in the development of gene-mediated therapies and the challenges that come along with treating rare neuromuscular disease. [WATCH TIME: 3 minutes]

Zolgensma Reinforces Efficacy in SPR1NT Results of Patients With SMA and 3 Copies of SMN2

March 19, 2022

The gene therapy from Novartis showed that it was both efficacious and well-tolerated in presymptomatic patients with SMA with 3 copies of SMN2, with no patients experiencing treatment-related serious adverse events.

Initial Phase 4 RESPOND Results, Omaveloxolone Continues to Perform in FA, Wearable Sensors Safe for FSHD

March 19, 2022

Neurology News Network for the week ending March 19, 2022.

Assessing the Growing Treatment Landscape for Myasthenia Gravis, Next Steps in Optimization: Henry Kaminski, MD

March 17, 2022

The chairman of the department of neurology at George Washington University discussed the reasons to be optimistic, but cautiously, about the expanding myasthenia gravis treatment toolbox. [WATCH TIME: 3 minutes]

Initial Safety Findings From Phase 4 RESPOND Study of Nusinersen Announced

March 17, 2022

After a mean follow-up of 64 days, the most common adverse events were infections and vomiting, with no deaths or reports of post-lumbar puncture syndrome.