Neuromuscular

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending September 1, 2023.

A panel of former AUPN presidents detailed how advances in neurology have changed the way the clinical specialty is taught, and why collaboration among leadership remains vital to creating an effective neurology department. [WATCH TIME: 7 minutes]

Despite pamrevlumab being generally safe and well tolerated, the potential first-in-class connective tissue growth factor inhibitor antibody did not meet its primary endpoint in a phase 3 trial for ambulator Duchenne muscular dystrophy.

Social determinants of health are conditions that people are born into and live under that affect their health, and can greatly impact clinical outcomes as well as contribute to health disparities and inequalities.

Findings from a real-world study among 120 patients with SMA suggests that nusinersen administration is safe and well tolerated, with only 30% of the patients mostly experiencing mild adverse events.

Former presidents of the AUPN shared perspective on the advances in neurology and how it was taught during their tenure.

The chief medical officer at Avidity Biosciences discussed the phase 1/2 EXPLORE44 trial, assessing an investigational treatment for Duchenne muscular dystrophy, and highlighted the potential of the treatment based on the recent FDA orphan drug and fast track designations. [WATCH TIME: 4 minutes]

The group of experts provided perspectives on the shift in how residency programs are developed and laid out, as well as efforts to increase the number of young neurologists in the field.

Former presidents of the AUPN shared perspective on the advances in neurology and how it was taught during their tenure.

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.