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 29th 2025
Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending August 29, 2025.
Alnylam Reports Positive Data on Patisiran in APOLLO-B Trial of ATTR Amyloidosis With Cardiomyopathy
August 4th 2022The Alnylam Pharmaceuticals agent, already approved for the treatment of polyneuropathy in those with hATTR amyloidosis, has now shown promise as a therapeutic option for patients with ATTR amyloidosis with cardiomyopathy.
Complexities With SMN2 Copies in Spinal Muscular Atrophy: Basil Darras, MD
August 1st 2022The associate neurologist-in-chief at Boston Children’s Hospital discussed the 4-copy conundrum and whether treatment decisions differ based on SMN2 copies for patients with spinal muscular atrophy. [WATCH TIME: 2 minutes]
Mechanistic Potential of Taldefgrobep Alfa in Spinal Muscular Atrophy: Lindsey Lee Lair, MD
July 28th 2022The vice president of clinical development at Biohaven shed light on a newly initiated phase 3 study evaluating a promising agent for patients with all types of spinal muscular atrophy. [WATCH TIME: 2 minutes]
Assessing Treatment Options and Improving Utilization for Spinal Muscular Atrophy: Basil Darras, MD
July 28th 2022The associate neurologist-in-chief at Boston Children’s Hospital provided insight on the treatment decisions clinicians make for patients with SMA and the challenges with improving optimization. [WATCH TIME: 3 minutes]
Prescribing Psychical Activity Regimens in Spinal Muscular Atrophy: Jacqueline Montes, PT, EdD
July 26th 2022The associate professor of rehabilitation and regenerative medicine at Columbia University Medical Center discussed the ways clinicians go about properly prescribing exercise regimens for patients with spinal muscular atrophy. [WATCH TIME: 3 minutes]
European Commission Passes Eladocagene Exuparvovec as First Approved Treatment for AADC Deficiency
July 22nd 2022In previous clinical studies, patients with AADC deficiency treated with the recombinant AAV2-based gene therapy achieved motor milestones, including head control and unassisted sitting.
Treating Familial ALS at the Source: The Introduction of Gene Therapies
July 20th 2022As scientific researchers continue to uncover new genetic links to amyotrophic lateral sclerosis, the rise of gene therapies to treat the neuromuscular disease will remain a development to watch in the coming years.
NeuroVoices: Lindsey Lee Lair, MD, on the Phase 3 RESILIENT Trial of Taldefgropeb Alfa in SMA
July 20th 2022The vice president of clinical development at Biohaven provided insight on a new phase 3 study assessing a muscle-targeted agent as an adjunct therapy to currently approved spinal muscular atrophy medications.
Muscle-Directed Therapy Apitegromab Reports Positive 24-Month Results in Spinal Muscular Atrophy
July 12th 2022Over a 2-year period, apitegromab-treated patients demonstrated sizable and sustained improvements in Hammersmith Functional Motor Scale-Expanded and substantial increases in Revised Upper Limb Module scores.