Neuromuscular

As part of our monthly clinician spotlight, NeurologyLive® highlighted expert Gary Hisch, MD, pediatric neurologist at Cleveland Clinic, who oversees programs for neuromuscular disorders such as for patients with spinal muscular atrophy.

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 August 26, 2023. [WATCH TIME: 3 minutes]

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending August 25, 2023.

TSHA-102, an adeno-associated virus vector-based gene therapy for Rett Syndrome, has also received Orphan Drug and Rare Pediatric Disease designations from the FDA.

Over a 3-treatment cycle, no new clinically significant safety observations were found, with significant dose-dependent increases in frataxin in muscle mRNA expression.

In a clinical study diagnosing genetic disorders, genomic sequencing did not report 19 variants found by a targeted neonatal gene-sequencing test and the targeted panel did not report 164 variants recognized by genomic sequencing.

Over a 28-day period, no adverse events led to study drug withdrawal, with decreased appetite as the most common treatment-related AE observed in the highest dosed cohort.

Over a 48-week stretch, a composite end point analysis suggested a survival treatment benefit with edaravone, further supporting its use in patients with ALS.

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.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending August 18, 2023.

Over the 36-month treatment period, nonambulatory patients with SMA treated with apitegromab showed improvements in patient-reported outcomes of daily living and fatigue.

AOC 1044, an exon 44-targeting agent, holds potential to fill a major unmet need for patients with Duchenne muscular dystrophy, as there are currently there are no approved therapies approved targeting this mutation.

At 6 weeks post-treatment, the patient saw clinical improvements in multiple domains, including autonomic function, vocalization, gross motor skills, and fine motor skills and hand function.

In a phase 3 study and open-label extension, patients treated with daxibotulinumtoxinA showed significant improvements in their condition, explained by changes on the Toronto Western Spasmodic Torticollis Rating Scale.

The associate professor of pediatrics in neurology and epilepsy at Northwestern University Feinberg School of Medicine discussed the FDA-cleared phase 1/2 trial of an exon 50 skipping therapy to address the specific mutations causing Duchenne muscular dystrophy. [WATCH TIME: 6 minutes]

The undergraduate program director at the University of South Carolina discussed the shift in teaching strategies for those in neurology and why more hands-on experience serves students better. [WATCH TIME: 5 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 general neurology.

The majority of patients with ALS lack a monogenic mutation, with only 15-20% of patients having a disease that is driven and controlled by a single gene.

Neurology News Network for the week ending August 12, 2023. [WATCH TIME: 3 minutes]

Since it was first discovered in the early 1900s, the therapeutic approaches to treating myasthenia gravis have changed significantly, with several proven treatment options.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending August 11, 2023.

BridgeBio Pharma met with the FDA to discuss the use of glycosylated α-dystroglycan levels as a surrogate end point for its phase 3 trial FORTIFY investigating BBP-418 and believes there is the potential for an accelerated approval.

The double-blind, placebo-controlled trial assesses change from baseline in the weekly average of daily self-reported worst pain intensity scores after 14 weeks of treatment.

Catch up on any of the neurology news headlines you may have missed over the course of July 2023, compiled all into one place by the NeurologyLive® team.

Results showed that 67% of treated patients in Stage A demonstrated evidence of clinical improvement, indicating that IgG autoantibodies play a significant role in the underlying biology of CIDP.

The trial is expected to include 14 individuals with genetically confirmed limb-girdle muscular dystrophy who will be randomly assigned 1:1 to either AB-1003 or placebo for a year-long treatment period.