The NeurologyLive® neuromuscular disease clinical focus page offers updates and information related to the clinical care and management of individuals with neuromuscular diseases such as amyotrophic lateral sclerosis, Duchenne muscular dystrophy, cerebral palsy, and spinal muscular atrophy, as well as rare diseases. This page consists of news, podcasts, and videos about the latest FDA actions, clinical guideline updates, interviews with physicians, study and clinical trial findings, and more.
August 4th 2025
Here's some of what is coming soon to NeurologyLive® this week.
Significance of Relaunched Patient Registry for Myasthenia Gravis Research: Richard Nowak, MD, MS
February 7th 2023The assistant professor of neurology at Yale School of Medicine spoke about the importance of the relaunch of the patient registry for research in myasthenia gravis. [WATCH TIME: 3 minutes]
Sanofi Discontinues Phase 3 Study of Tolebrutinib in Myasthenia Gravis
February 7th 2023The trial was originally placed on hold because of reported cases of drug-induced liver injury in patients who received the study drug, potentially caused by a preexisting factors related to hepatic dysfunction.
National Women Physicians Day: 10 Moments That Matter in Medicine
February 3rd 2023The list of women's contributions to the field of medicine is a long one, encompassing the hard work of hundreds of individuals. To highlight some of these pioneers, NeurologyLive® has compiled a timeline of 10 of the moments that matter in the history of women in medicine.
The expert panel comments on ideal patient profiles for SMA treatment with nusinersen, onasemnogene abeparvovec, and risdiplam. O painel de especialistas comenta os perfis ideais de pacientes para tratamento da atrofia muscular espinhal com nusinersena, onasemnogeno abeparvoveque e risdiplam.
Drs Rodrigo de Holanda Mendonça and Adriana Banzzatto Ortega discuss clinical trial data with nusinersen, onasemnogene abeparvovec, and risdiplam. Drs Rodrigo de Holanda Mendonça e Adriana Banzzatto Ortega discutem dados de testes clínicos com nusinersena, onasemnogeno abeparvoveque e risdiplam.
NeuroVoices: Rachel Alvarez on Identifying Key Treatment Needs for Congenital Muscular Dystrophies
February 1st 2023The executive direct of Cure CMD discussed the state of care for patients with congenital muscular dystrophies, the need for new treatments, and the patience with gene therapies.
Mission Behind Cure CMD’s Research Roundtable, Efforts Around CMDs: Rachel Alvarez
January 28th 2023The executive director of Cure CMD discussed efforts being done to bring the stakeholder community together and improve the care for patients with congenital muscular dystrophies. [WATCH TIME: 4 minutes]
Drs Juliana Gurgel Giannetti and Rodrigo de Holanda Mendonça provide an overview of available treatments for spinal muscular atrophy in Brazil. / Drs Juliana Gurgel Giannetti and Rodrigo de Holanda Mendonça discutem tratamentos para atrofia muscular espinhal disponíveis no Brasil.
Drs Juliana Gurgel Giannetti and Rodrigo de Holanda Mendonça discuss genetic changes in spinal muscular atrophy and genetic screening in Brazil. / Drs Juliana Gurgel Giannetti and Rodrigo de Holanda Mendonça discutem mudanças genéticas em atrofia muscular espinhal e screening genético no Brasil.
Drs Juliana Gurgel Giannetti, Adriana Banzzatto Ortega and Marcelo Kerstenetzky discuss symptoms of spinal muscular atrophy and diagnosis and referral process in Brazil. / Drs Juliana Gurgel Giannetti, Adriana Banzzatto Ortega e Marcelo Kerstenetzky discutem sintomas da atrofia muscular espinhal, diagnóstico e processo de encaminhamento no Brasil.
Overview of Spinal Muscular Atrophy (SMA) / Visão geral sobre Atrofia Muscular Espinhal (AME)
Dr Rodrigo de Holanda Mendonça provides an overview of spinal muscular atrophy (SMA). / Dr Rodrigo de Holanda Mendonça dá uma visão geral sobre Atrofia Muscular Espinhal (AME).
MDA’s Focus on Better Access for Patients With Neuromuscular Disorders: Paul Melmeyer
January 25th 2023Ahead 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]