Muscular Dystrophy Association Clinical and Scientific Conference (MDA)

The professor of neurology and pediatrics at University of Rochester Medical Center shared her thoughts on the 2023 MDA Clinical and Scientific Conference, and the progress made in the treatment of neuromuscular disorders.

The palliative care physician and assistant professor of medicine at Johns Hopkins Bayview Medical Center provided perspective on current state of end-of-life care for neuromuscular disorders and areas to improve upon. [WATCH TIME: 4 minutes]

The chief medical officer at ML Bio Solutions spoke about the advances in neuromuscular diseases such as limb girdle muscular dystrophy at the 2023 MDA Conference. [WATCH TIME: 3 minutes]

Efgartigimod-treated patients consistently outperformed placebo regardless of concomitant medication, thymectomy status, and timing of diagnosis.

After previously showing positive effects on MG-ADL and QMG, ravulizumab continued to show improvements in ocular and respiratory muscle domains in myasthenia gravis.

The director of the Center for Biologics Evaluation Research at the FDA provided an overview of the Keynote Address he gave at the 2023 Muscular Dystrophy Association Clinical and Scientific Conference. [WATCH TIME: 3 minutes]

More than 90% of patients, ambulatory and non-ambulatory, experienced improvements in Medical Research Council score within 6 months of treatment.

Differences in baseline characteristics and eligible criteria in presymptomatic infants with spinal muscular atrophy led to differential results on motor and nonmotor clinical outcomes.

Test your neurology knowledge with NeurologyLive®'s weekly quiz series, featuring questions on a variety of clinical and historical neurology topics. This week's topic is the history of the Muscular Dystrophy Association (MDA).

The associate professor of neurology at Columbia University and medical consultant and care center director for MDA offered his insight into the latest progress in ALS treatment and some of what will be discussed at the upcoming MDA Clinical & Scientific Conference.

Stanley Appel, MD, shared his insight into the MDA session track on amyotrophic lateral sclerosis, the latest science advances to benefit patients, and where the field is headed with research and treatment.

Ahead of the 2023 MDA Conference, which will be held from March 19-22, 2023, in Dallas, Texas, the vice president of public policy and advocacy at the Muscular Dystrophy Association provided an overview of the current state of access and the need for more advocates in neuromuscular disease. [WATCH TIME: 14 minutes]

Angela Lek, PhD, and Tahseen Mozaffar, MD, offered their insight into myositis and the clinical needs for the population, ahead of a focused session on it, chaired by Mozzafar at the MDA’s Clinical & Scientific Conference.

The president and CEO, as well as the executive vice president and chief research officer, of MDA, together offered an in-depth overview of what to expect from the 2023 MDA Conference, which will be held from March 19-22, 2023, in Dallas, Texas. [WATCH TIME: 12 minutes]

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]

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 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]

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.

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.

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]

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.

Neurology News Network for the week ending March 19, 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]

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.

Jinsy Andrews, MD, MSc, FAAN, offered her perspective on the ongoing therapeutic boom in neuromuscular disease and some of the critical needs for physicians to keep up with a rapidly progressing treatment paradigm.

The president and CEO of the Muscular Dystrophy Association spoke about the highlights of this year’s annual meeting and the recent therapeutic advances for neuromuscular disorders. [WATCH TIME: 3 minutes]

The chairman of the department of neurology at George Washington University discussed his presentation at MDA 2022 and the value of hearing patient perspective at the conference. [WATCH TIME: 2 minutes]

The director of neuromuscular clinical trials at Columbia University Irving Medical Center shared her perspective on the challenges of familiarizing oneself with the influx of novel medications for neuromuscular diseases. [WATCH TIME: 3 minutes]

Updates from the open-label, delayed-start period of the pivotal 2-part MOXIe trial (NCT02255435) support previous positive primary end points findings from part 2 of the study.

Vamorolone, an investigational agent for Duchenne muscular dystrophy, showed an initial disease-modifying effect that was maintained over a follow-up period of 48 weeks.