News

The chief of neurology and co-director of the Neurological Institute at the Children’s Hospital Los Angeles described some of the major resource and policy-level barriers for gene therapy in pediatric neurological conditions. [WATCH TIME: 6 minutes]

The associate professor of neurosurgery at Robert Wood Johnson Medical School talked about minimally invasive techniques and neurostimulation strategies to make epilepsy surgery safer and more effective. [WATCH TIME: 7 minutes]

The executive vice president and chief research officer of the Muscular Dystrophy Association shared her reaction to the recent FDA approval of givinostat for Duchenne muscular dystrophy.

The first patient enrolled into the phase 1/2a ArthemiR trial assessing ATX-01 myotonic dystrophy type 1 is expected in the second quarter of 2024.

The new position statement proposes the use of CGRP-targeting therapies to be used as first-line treatments, eliminating the need to fail multiple medications before access.

The director of Dysautonomia Clinic discussed neurological manifestations of Long COVID, such as headache, cognitive complaints, and sleep problems, and how they are similar to those seen with migraine comorbidities. [WATCH TIME: 5 minutes]

The newly initiated phase 3 trial follows a successful phase 2 study where treatment with pitolisant resulted in greater improvement on the Epworth Sleepiness Scale for Children and Adolescents than placebo.

Sleeping Around the Podcast × NeurologyLive brings you a clinical overview of a newly published study highlighting extracellular vesicle-associated miRNA features that could be diagnostically informative of RBD in patients with Parkinson disease.

The newly cleared i-STAT TBI test can be used to help assess patients up to 24 hours after injury at patient bedside, with lab-quality results produced in 15 minutes.

Therapeutic effects were noticeable within 15–20 days post-injection, with five of six patients achieving complete pain relief.

Ganaxolone, a synthetic neurosteroid, has ongoing studies in tuberous sclerosis complex and super refractory status epilepticus, as well as upcoming research in Lennox-Gastaut syndrome and other epileptic encephalopathies.

The professor of neurology at Johns Hopkins Medicine discussed the trial design of the phase 4 RENEW study assessing the long-term safety profile of mitoxantrone in multiple sclerosis. [WATCH TIME: 9 minutes]

SPN-830, an apomorphine infusion device, had its new drug application submitted using data from the phase 3 TOLEDO trial.

The chief of neurology and co-director of the Neurological Institute at the Children’s Hospital Los Angeles discussed the hesitancy to enroll children with neurodevelopmental issues in early intervention trials without proven products or markers. [WATCH TIME: 5 minutes]

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

The presence of a sleep disorder was associated with a nearly 2-fold increase in the risk of increased health care utilization, with specific odds associated with individual sleep disorders.

The assistant professor of neurology at Johns Hopkins Medicine talked about her preclinical research in immunology to explore the intricate mechanisms controlling chronic inflammation in the central nervous system. [WATCH TIME: 5 minutes]

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 on limb girdle muscular dystrophy.

The phase 1 study, dubbed PRO-101, assesses the safety, tolerability, pharmacokinetics, and pharmacodynamics of prosetin in both patients with ALS and healthy volunteers.

A recent study indicated that certain antiseizure medications used in pregnant women with epilepsy had a lower risk of major congenital malformations.

Neurology News Network. for the week ending April 6, 2024. [WATCH TIME: 3 minutes]

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

A recent published letter affirmed the safety of cenobamate, an FDA-approved antiseizure medication, in a large global patient population.

The executive medical lead at Italfarmaco SpA expressed his confidence in givinostat to mitigate the progression of Duchenne muscular dystrophy, emphasizing its multifactorial mechanism of action. [WATCH TIME: 5 minutes]

Mind Moments®, a podcast from NeurologyLive®, brings you an interview with Francesco Saccà, MD, PhD. [LISTEN TIME: 18 minutes]

Findings from a recent study revealed the significant influence of lysosomal polygenic burden on cognitive decline in patients Parkinson disease who also have a low risk of Alzheimer disease.

Although results from the phase 2 LixiPark trial showed that lixisenatide may slow motor symptom progression in early Parkinson disease, reported gastrointestinal adverse effects raise some safety considerations.

Amylyx Pharmaceuticals’ AMX0035, despite its safety profile, did not show significant efficacy among patients with amyotrophic lateral sclerosis in the phase 3 PHOENIX trial.

The professor of neurology at Johns Hopkins Medicine discussed the limited impact of current therapies on retinal atrophy rates in progressive multiple sclerosis at the 2024 ACTRIMS Forum. [WATCH TIME: 5 minutes]

TUDCA, a hydrophilic bile acid that is normally produced endogenously in humans in the liver, failed to slow ALS disease progression in comaprison with placebo.