Neuromuscular

The 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]

The 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]

The associate neurologist-in-chief at Boston Children’s Hospital discussed the importance of defining and acting on spinal muscular atrophy in its presymptomatic stages. [WATCH TIME: 4 minutes]

In addition to reductions of SOD1 protein, earlier start tofersen demonstrated positive effects in clinical function, respiratory function, muscle strength, and quality of life in patients with SOD1 ALS.

The 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]

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 headache and migraine.

Neurology News Network for the week ending July 23, 2022. [WATCH TIME: 4 minutes]

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending July 22, 2022.

The associate professor of rehabilitation and regenerative medicine at Columbia University Medical Center provided insight on the tools and gaps in care for assessing physical capacity in patients with spinal muscular atrophy. [WATCH TIME: 4 minutes]

In previous clinical studies, patients with AADC deficiency treated with the recombinant AAV2-based gene therapy achieved motor milestones, including head control and unassisted sitting.

Treatment with CNM-Au8 was well-tolerated, with no new safety signals, and decreased the risk of death by 70% compared with placebo on unadjusted Kaplan-Meier analyses.

As 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.

The 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.

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 neuromuscular disorders.

Neurology News Network for the week ending July 16, 2022. [WATCH TIME: 4 minutes]

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending July 15, 2022.

The new phase 2b trial expands on successful phase 2a findings, in which treatment with SCI-110 resulted in a 21% reduction in tics across a cohort of adults with Tourette syndrome.

Over 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.

Taldefgrobep alfa, a muscle-targeted experimental treatment potentially used in combination with other SMA therapies, will be evaluated in a cohort of 180 patients with SMA, regardless of ambulatory status or disease classification.

Here's what is coming soon to NeurologyLive®.

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 stroke and cerebrovascular disease.

Neurology News Network for the week ending July 9, 2022. [WATCH TIME: 4 minutes]

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending July 8, 2022.