Neuromuscular

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

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 FDA has reversed course, deciding to hold an advisory committee meeting ahead of the May 29, 2023, action date for Sarepta’s investigational DMD agent SRP-9001 (also known as delandistrogene moxeparvovec).

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.

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.

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.

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

Disease pathogenesis is attributed to oxidative stress—which can be regulated by NRF2, which, in turn, binds to antioxidant responsive elements in the promoter of the target gene FXN to control its expression.

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.

The Reata Pharmaceuticals therapy, branded as Skyclarys, is indicated for adults and adolescents aged 16 years and older.

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.

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.