Neuromuscular

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.

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

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending February 24, 2023.

Drs Juliana Gurgel Giannetti, Rodrigo de Holanda Mendonça and Marcelo Kerstenetzky examine barriers to participation in clinical trials in Brazil for patients with spinal muscular atrophy. Drs Juliana Gurgel Giannetti, Rodrigo de Holanda Mendonça e Marcelo Kerstenetzky analisam barreiras para a participação de pacientes com atrofia muscular espinhal em testes clínicos no Brasil.

The expert panel discusses challenges in clinical monitoring and access to care for patients with spinal muscular atrophy in Brazil. O painel de especialistas discute desafios no monitoramento clínico e no acesso a atendimento de saúde para pacientes com atrofia muscular espinhal.

To honor Black History Month, NeurologyLive® spoke with influential Black clinicians on the leaders they look up to, the ongoing fight to overcome racial disparities, and ways to encourage diversity in health care.

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.

To honor Black History Month, NeurologyLive® spoke with influential Black clinicians on the leaders they look up to, the ongoing fight to overcome racial disparities, and ways to encourage diversity in health care.

To honor Black History Month, NeurologyLive® spoke with influential Black clinicians on the leaders they look up to, the ongoing fight to overcome racial disparities, and ways to encourage diversity in health care.

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

The lead of the Myasthenia Gravis Foundation of America Global Myasthenia Gravis Patient Registry spoke on 2 additional attributes of the patient registry. [WATCH TIME: 3 minutes]

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