Neuromuscular

Transgender and gender-diverse patients have unique neurological considerations, but often, neurologists are unaware of the needs that are specific to this community.

Neurology News Network for the week ending March 23, 2024. [WATCH TIME: 3 minutes]

A patient advocate within the ALS community discussed the importance of incorporating patients and their voices into clinical meetings and trial design to ultimately meet treatment goals. [WATCH TIME: 4 minutes]

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending March 22, 2024.

The chief medical officer of NMD Pharma provided perspective on a newly launched phase 2 study assessing NMD670, a first-in-class neuromuscular transmission enhancer, in patients with spinal muscular atrophy type 3. [WATCH TIME: 5 minutes]

Branded as Duvyzat, givinostat is the first nonsteroidal drug approved to treat patients with all genetic variants of DMD.

The professor of pediatric neuromuscular diseases at the University of Oxford provided commentary on the commonly used scales to assess neuromuscular function and where inconsistencies have been observed. [WATCH TIME: 7 minutes]

This complex waste clearance pathway within the CNS plays a crucial role in maintaining brain homeostasis, as a mechanism of protein waste removal alongside autophagy and protein aggregate ubiquitination.

The chief executive officer at Elixirgen Therapeutics provided commentary on the promise of the Bobcat mRNA, and the mechanism of action for mRNA-like therapies to treat Duchenne muscular dystrophy.

Despite the overall cohort not reaching statistical significance on the primary end point, a subgroup of patients on SLS-005 without AMX0035 performed better.

Join us on April 8 on our LinkedIn and X pages for our #PreAANChat premeeting social chat ahead of the 2024 American Academy of Neurology Annual Meeting!

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 Purple Day and epilepsy awareness.

Neurology News Network for the week ending March 16, 2024. [WATCH TIME: 3 minutes]

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending March 15, 2024.

Over 61.1 patient-years of treatment experience, AOC 1001 was well tolerated in patients with DM1, with nausea and headache the most commonly reported adverse events.

Monepantel, a novel, potent, inhibitor of the mTOR pathway, was safe in patients with ALS, resulting in stabilized ALS Functional Rating Scale scores.

The delivery device is prefilled with the prescribed dose of Acthar Gel in 40- or 80-unit versions, and may help administration of the appropriate subcutaneous dose of Acthar Gel.

Comprised of more than 400 patients with SMA, results showed that wanting to be treated with all possible DMTs was the most common reason for combination therapy.

Amanda Peltier, MD, a professor of neurology at Vanderbilt University Medical Center, spoke on current care needs for adults with neuromuscular disorders and the realistic ways to enhance clinical care going forward.

The instructor in the department of cell biology at Emory University provided context on a presentation at MDA 2024 regarding mutations in Kif5a in familial forms of amyotrophic lateral sclerosis. [WATCH TIME: 4 minutes]

Here's some of what is coming soon to NeurologyLive® this week.

Catch up on any of the neurology news headlines you may have missed over the course of February 2024, compiled all into one place by the NeurologyLive® team.

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 Purple Day and epilepsy awareness.

These finding suggest that even immune cells in the brain can play a significant role in the progression of neurodegenerative diseases like FA.

Neurology News Network for the week ending March 9, 2024. [WATCH TIME: 3 minutes]

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending March 8, 2024.

Over the coming weeks, Amylyx will undergo heavy discussions with regulators and community members to decide the next best steps for AMX0035 after the agent failed to meet its primary end point in its latest study.

The real-world, 3-year study is expected to include 30 males with SMA on risdiplam, an SMN2 splicing modifier, to assess fertility-related outcomes.

Findings from the recent phase 3b SMART trial affirm the safety and efficacy of intravenous onasemnogene abeparvovec (Zolgensma; Novartis) in spinal muscular atrophy when patient weights range from 8.5 kg to 21 kg.