Neuromuscular

The director of the ALS Clinic at Allegheny Health Network provided perspective on the advancements in the treatment and management of adult-onset spinal muscular atrophy.

The president of the ANA and the chair of the annual meeting programming committee provided perspective on the upcoming ANA Annual Meeting and what clinicians may get out of the meeting. [WATCH TIME: 7 minutes]

The chief medical officer at Avidity Biosciences talked about how its exon skipping AOC therapies are designed to address the lack of functional dystrophin protein among patients with Duchenne muscular dystrophy to improve muscle function. [WATCH TIME: 4 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 multiple sclerosis.

Findings from an open-label extension trial among 45 patients with ALS showed a significant median survival benefit of 19.3 months and a significant 52% decreased risk of ALS clinical worsening events after treatment.

A group of esteemed panelists discussed the important values neurology department directors and educators should prioritize when teaching students of the future. [WATCH TIME: 6 minutes]

A trio of former and current neurology department directors discussed ways in which residency and fellowship programs may change as technology and therapeutic capabilities continue to expand. [WATCH TIME: 8 minutes]

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.