Neuromuscular

The director at the Foundation for the National Institutes of Health provided commentary on a recently launched partnership aimed at building a repository of ALS data to advance drug development for the disease.

Initial data from a few pediatric and adult patients with Rett syndrome showed that treatment with TSHA-102 resulted in numerous positive enhancements across multiple efficacy measures and clinical domains.

The cleared phase 1/2 clinical trial will assess SPG302 as a once-a-day pill among patients with amyotrophic lateral sclerosis.

Key opinion leaders (KOLs) address the challenges and obstacles associated with transitioning patients from pediatric to adult care as they age out of the pediatric system, offering valuable insights and strategies for successfully navigating this process.

Leading medical professionals specializing in spinal muscular atrophy (SMA) discuss the common clinical presentations of undiagnosed or newly diagnosed adult patients, highlighting crucial signs and symptoms that healthcare providers should recognize.

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

Neurology News Network. for the week ending June 15, 2024. [WATCH TIME: 4 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 narcolepsy.

Rakesh Jain, PhD, MS, clinical professor of psychiatry at the Texas Tech University School of Medicine, provided insight on a recently approved tablet dosage for deutetrabenazine, an FDA-approved therapy for chorea and tardive dyskinesia associated with Huntington disease.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending June 14, 2024.

AOC 1020 (del-brax) from Avidity Biosciences shows over 50% reduction in DUX4 regulated genes, trends of functional improvement, and favorable safety in FSHD patients.

Satellos Bioscience has established a clinical advisory board to aid in developing their lead drug candidate SAT-3247, an oral small molecule therapy for Duchenne muscular dystrophy.

Fordadistrogene movaparvovec is a recombinant AAV9 agent carrying a shortened version of the dystrophin gene, being assessed in the phase 3 CIFFREO study.

For up to 24 months, patients on mirdametinib demonstrated significant improvements in pain severity, pain interference, and health-related quality of life, in addition to deep and durable plexiform neurofibroma volume reductions.

Although a small sample of 5 patients with DMD, results showed significant expression of microdystrophin and reductions in creatine kinase, a biomarker of muscular distress, in GTN0004-treated patients.

Deflazacort, a corticosteroid approved for children with Duchenne muscular dystrophy, has been on the market since 2017 under the name Emflaza.

John Novak, MD, MS, director of the OhioHealth ALS Clinic and vice chair of the ALS Association Care Services Committee, provided insight on the upcoming ALS Nexus Conference, the first ever educational event from the organization, spanning multiple days in July.

The FIREFISH study showed that most children maintained or improved motor functions and feeding abilities over five years.

Panelists examine the natural history of spinal muscular atrophy (SMA), assessing the characteristic indicators of motor function deterioration and the loss of ambulatory abilities as patients advance in age.

Key opinion leaders offer a concise summary of the clinical characteristics and classifications of spinal muscular atrophy (SMA), emphasizing the distinctive features observed in adult patients with SMA.

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

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

Neurology News Network. for the week ending June 9, 2024. [WATCH TIME: 4 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 stroke and large hemispheric infarction.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending June 7, 2024.

Biogen's tofersen, an antisense oligonucleotide, remains the first and only marketed therapy to treat patients with SOD1-mutated amyotrophic lateral sclerosis.

Glioblastoma, a highly aggressive brain cancer, presents substantial treatment challenges necessitating innovative therapies and interdisciplinary research to improve patient outcomes.

The FDA approval of the increased maximum daily dose for amifampridine offers clinicians and patients greater flexibility in treatment regimens for the management of Lambert-Eaton myasthenic syndrome.

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 on amyotrophic lateral sclerosis (ALS).