Neuromuscular

The chief executive officer at Elixirgen Therapeutics discussed the company’s mRNA as a promising therapeutic avenue for Duchenne muscular dystrophy. [WATCH TIME: 6 minutes]

Over the 24-week treatment period, satralizumab demonstrated a well tolerated safety profile that was consistent with its previous indication.

The professor of neurology at Vanderbilt University Medical Center discussed advocating for reinstating personalized resources, expanding genetic testing, and improving transitional care in muscular dystrophy. [WATCH TIME: 5 minutes]

The second-year PhD student in bioinformatics at Boston University who lives with LAMA2 congenital muscular dystrophy talked about the potential impact and challenges of gene therapy in neuromuscular diseases. [WATCH TIME: 5 minutes]

The neuromuscular specialist at the Neurology & Neuromuscular Care Center discussed the pressing need to address the transition of care from pediatric to adult healthcare systems in Duchenne muscular dystrophy. [WATCH TIME: 6 minutes]

The professor of neurology at Vanderbilt University Medical Center talked about the importance of multidisciplinary care centers to improve accessibility and quality of care for patients with neuromuscular disorders. [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 movement disorders and multiple system atrophy.

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.