Neuromuscular

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]

Topline data from the phase 3 ADAPT-SC study (NCT04735432) suggest that the subcutaneous formulation of the Argenx product was statistically noninferior to the recently approved IV formulation, efgartigimod afla-fcab (Vyvgart).

The president and chief executive officer of The ALS Association provided commentary on the most pertinent issues with managing patients with ALS and the ways to improve current treatment standards. [WATCH TIME: 4 minutes]

The president and chief executive officer of The ALS Association provided insight on how the organization is advancing the therapeutic pipeline of ALS agents for the foreseeable future. [WATCH TIME: 3 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]

The president and chief executive officer of The ALS Association expanded on the organization’s greatest goal—making ALS livable by 2030—and how it plans to work towards that over the next decade. [WATCH TIME: 3 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.

In terms of cardiomyopathy, disease-modifying bioactivity of cardiosphere-derived cells was shown by improvements in left ventricular ejection fraction, a commonly used measure of global heart function.

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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]

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 optic neuritis and neuromyelitis optica spectrum disorder (NMOSD).

Kaplan-Meier estimates showed a 61% decreased risk of death on CNM-Au8 compared with predicted median survival from a published European Network to Cure ALS model.

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.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending March 18, 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.

The division chief of neuromuscular disorders and vice-chair of research at Virginia Commonwealth University offered his insight into the advances in genetic approaches to LGMD. [WATCH TIME: 2 minutes]

The 2022 MDA National Ambassador and patient with Bethlem myopathy spoke to the critical need to improve diagnosis delays in neuromuscular disorders and effective communication between patients and their physicians. [WATCH TIME: 2 minutes]

Data may be useful when analyzing disease progression and treatment efficacy in future facioscapulohumeral muscular dystrophy clinical trials.

The treatment was well tolerated at 48 weeks in patients with DMD with a confirmed mutation amenable to exon 45 skipping.

Part B of the 2-part study will have patients previously on placebo receive SRP-9001 for an additional 52 weeks.