Neuromuscular

A new analysis of phase 3 MOVE-FA trial presented at MDA 2024 revealed promising outcomes for vatiquinone, an oral 15-lipoxygenase inhibitor, in patients with Friedreich ataxia.

Over a 52-week treatment period, treatment with SRP-9001 resulted in improvements in secondary outcomes of time to rise, micro-dystrophin expression, and 10-meter walk/run.

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

Patients with Duchenne muscular dystrophy between ages of 6 and 24 months old demonstrated a similar safety profile on eteplirsen than those between 24 and 48 months of age.

In a real-world study, eterplirsen was safe for patients with Duchenne muscular dystrophy, with sustained or improved status in function.

Tadalafil shows potential in addressing microvascular impairment in Duchenne muscular dystrophy, with post-contractile MRI as a screening tool.

Jeff Chamberlain, PhD, a leading professor in gene therapy focused on Duchenne muscular dystrophy, shared his reaction to being named the recipient of the 2024 MDA Legacy Award, as well as the state of the DMD field currently.

Catch up on any of the neurology news headlines you may have missed over the course of March 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 Alzheimer disease and dementia.

Neurology News Network for the week ending March 1, 2024. [WATCH TIME: 3 minutes]

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending March 1, 2024.

The chief medical advisor at the Muscular Dystrophy Association provided perspective on the upcoming meeting and the conversations surrounding new therapeutics for diseases that once had little to nothing. [WATCH TIME: 4 minutes]

The program director in the division of Neuroscience at the National Institute of Neurological Disorders and Stroke provided context on the importance of Rare Disease Day and strides made over the years. [WATCH TIME: 5 minutes]

Rare Disease Day raises awareness for rare disorders, highlighting challenges, improving detection, and fostering collaboration for treatment advancements.

In honor of Rare Disease Day, the vice president of public policy and advocacy at the Muscular Dystrophy Association shared his perspective of advocacy and policy progress in the realm of rare neuromuscular diseases. [WATCH TIME: 6 minutes]

Caroline Just, MD, reflects on the challenges of diagnosing and treating rare neurological diseases, urging for improved education, collaboration, and research efforts.

A recent study showed that the 2021 European guidelines for chronic inflammatory demyelinating polyradiculoneuropathy provided a clearer definition of variants for the disease.

ATH-1105 protected various cell culture systems from glutamate-mediated toxicity and its pathological alterations, which include mitochondrial dysfunction, apoptotic signaling, and TDP-43 mis-localization, among others.

The antisense oligonucleotide will continue to be assessed in a large-scale phase 3 study, CARDIO-TTRansform, which will have data read out in 2025.

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

Repetitive transcranial magnetic stimulation, a more widely used symptomatic treatment, was able to improve several outcomes for patients with cerebellar ataxia, especially at high frequencies.

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 sleep disorders and hypersomnias.

PepGen has begun its phase 2 CONNECT1-EDO51 trial, dosing the first patient with PGN-EDO51, an agent for Duchenne muscular dystrophy amendable to an exon 51 skipping.

Neurology News Network for the week ending February 24, 2024. [WATCH TIME: 3 minutes]

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending February 23, 2024.

The professor in the Department of Translational Neuroscience at Barrow Neurological Institute provided perspective on the approval of tofersen (Qalsody; Biogen) for patients with ALS and the ways neurofilament light will be used going forward. [WATCH TIME: 3 minutes]

A recent phase 3 trial demonstrated that N-acetyl-l-leucine significantly improved symptoms in patients with Niemann-Pick disease type C, showing promise as a potential treatment.

A recent observational study detected anti-DLAT antibodies in the serum of patients with chronic inflammatory demyelinating polyneuropathy with a sensory-dominant phenotype.

After a positive phase 1 study, DNL788, an RIPK1 inhibitor, failed to distinguish itself from placebo in HIMALAYA.

The associate professor of neurology at Columbia University talked about a track session that he will be chairing at the upcoming MDA conference on amyotrophic lateral sclerosis genetic research. [WATCH TIME: 5 minutes]