Neuromuscular

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending March 17, 2023.

Ahead of the 2023 MDA Clinical and Scientific Conference, Anne Connolly, MD, provided perspective on conversations surrounding holistic approaches to neuromuscular diseases.

Jeffery Neul, MD, PhD, director of the Vanderbilt Kennedy Center, and professor of pediatrics at Vanderbilt University Medical Center, shared his reactions and thoughts to the approval of trofinetide.

In honor of Brain Awareness Week, a group of experts in the care of patients with neurological conditions—Ronald C. Petersen, MD, PhD; Brian Grosberg, MD, FAHS; James Beck, PhD; Michael Levy, MD, PhD; Stanley H. Appel, MD —shared their perspectives on hot topics of treatment and management in neurology.

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Jeffrey L. Neul, MD, PhD. [LISTEN TIME: 12 minutes]

Here's some of what is coming soon to NeurologyLive® this week.

The therapy, marketed as Daybue, is the first therapy indicated for Rett syndrome with supporting data from the pivotal phase 3 LAVENDER study.

Neurology News Network for the week ending March 11, 2023. [WATCH TIME: 3 minutes]

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending March 10, 2023.

Treatment with a lower dose of CNM-Au8 resulted in significantly decreased risk of death, feeding tube placement, assisted ventilation, and all-cause hospitalization, among other survival outcomes.

Batoclimab, a fully human anti-FcRn monoclonal antibody, has demonstrated an ability to significantly alleviate patients’ symptoms and improve quality of life in myasthenia gravis.

Efgartigimod resulted in rapid impact on health-related quality of life, with improvements that were consistent across multiple measures and were similar for the effect seen across 2 treatment cycles.

Here's some of what is coming soon to NeurologyLive® this week.

With an anticipated life expectancy of 3 years from the time of symptom onset, an effective treatment strategy is essential in ALS—and recent therapeutic progress has built a foundation of hope for the community.

Neurology News Network for the week ending March 4, 2023. [WATCH TIME: 3 minutes]

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending March 3, 2023.

The director of the Ann Kimball & John W. Johnson Center for Cellular Therapeutics at Houston Methodist provided insight on previously approved therapies for ALS, and whether they can work synergistically with agents that enhance regulatory T cells function. [WATCH TIME: 5 minutes]

The expert panel shares clinical pearls for treatment and management of Spinal Muscular Atrophy. O painel de especialistas compartilha recomendações valiosas para o tratamento e manejo da atrofia muscular espinhal.

Dr Marcelo Kerstenetzky highlights areas of unmet needs in rare diseases education for healthcare professionals in Brazil. Dr Marcelo Kerstenetzky ressalta áreas de carência na educação sobre doenças raras para profissionais de saúde no Brasil.

The expert panel shares recommendations and advice for care teams and families of patients with spinal muscular atrophy. O painel de especialistas compartilha recomendações e conselhos para equipes de saúde e familiares de pacientes com atrofia muscular espinhal.

Three of 4 patients withdrew from their previous enzyme replacement therapy following treatment with AT845 gene therapy.

A group of experts in the care of patients with rare diseases—Emma Ciafaloni, MD, FAAN; Martina Bebin, MD, MDA; Timothy Miller, MD, PhD; George Small, MD; Bruce Cree, MD, PhD, MAS, FAAN—shared their perspectives on hot topics of treatment and management.

In recognition of Rare Disease Day, the NeurologyLive® team offered an extensive update on the state of care and treatment for a few rare neurological diseases, including Pompe disease, Rett syndrome, among others.

In recognition of Rare Disease Day, the NeurologyLive® team offered an extensive update on the state of care and treatment for a few rare neurological diseases, including Pompe disease, Rett syndrome, among others.

In a dose-ranging study, the gene therapy achieved 78% Gb3 substrate clearance at 6 months and 77% reduction in urine podocyte loss in 1 of the first kidney biopsies.