Neuromuscular

Early treatment of CIDP, within 1-year of onset, is associated with better long-term outcomes, highlighting key prognostic factors and treatment timing.

The professor and chair of the Department of Molecular Pathobiology at NYU College of Dentistry discussed groundbreaking research on chronic pain, focusing on the discovery of new mechanisms involving nerve growth factor and its receptors.

NeurologyLive® will provide in-depth coverage of conferences in 2025, highlighting advancements in diagnostics, treatments, and multidisciplinary care across multiple neurological conditions.

Neurology News Network. for the week ending January 11, 2025. [WATCH TIME: 4 minutes]

The professor and chair of the Department of Molecular Pathobiology at NYU College of Dentistry provided a clinical overview of preclinical research identifying a new receptor for nerve growth factor that plays an important role in pain signaling. [WATCH TIME: 4 minutes]

The senior vice president of research at The ALS Association discussed the need for standardizing ALS diagnosis across institutions to reduce delays and ensure timely treatment. [WATCH TIME: 4 minutes]

Results from the open-label phase 1/2 study of tividenofusp alfa demonstrated positive effects on evidence-based surrogate end points in participants with Hunter syndrome.

SGT-212 delivers full-length frataxin via MRI-guided intradentate nucleus infusion and intravenous administration to target mitochondrial dysfunction in neurons and cardiomyocytes, addressing both neurologic and cardiac symptoms.

The senior vice president of research at The ALS Association provided commentary on recently published research that found specialists to diagnose ALS twice as fast as general neurologists. [WATCH TIME: 4 minutes]

Despite not meeting its primary and key secondary end points, an exploratory high dose cohort of fosigotifator demonstrated promising results on end points of muscle strength.

Over a 24-week period, DNL343 failed to distinguish itself from placebo on the primary end point of ALSFRS-R, as well as on other secondary end points of muscle strength and respiratory function.

Most participants showed improvements in motor function, with 61% achieving a clinically meaningful increase in HFMSE scores by week 52 and 41% achieving similar gains in RULM scores.

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

As part of NeurologyLive®'s Year in Review, we've compiled the most-watched expert-led video series that appeared on the website in 2024.

David Lynch, MD, PhD, a professor of neurology at the University of Pennsylvania Perelman School of Medicine, discussed the significance of a number of recently presented analyses highlighting omaveloxlone, the first approved therapy for Friedreich ataxia.

Neurology News Network. for the week ending January 4, 2025. [WATCH TIME: 4 minutes]

Imlifidase-IVIg treatment led to faster disability improvement (3 weeks) and earlier independent walking (6 weeks) compared to IVIg alone.

Novartis plans to present OAV101 IT data at a 2025 medical meeting and share results with regulatory agencies, including the FDA, to support approval for patients with SMA in need.

Capricor may qualify for a priority review voucher from the FDA if deramiocel receives marketing approval for the treatment of Duchenne muscular dystrophy.

As part of NeurologyLive®'s Year in Review, we've compiled the most-listened episodes of our biweekly podcast, Mind Moments®.

As part of NeurologyLive®'s Year in Review, we've compiled the most-watched interview clips that appeared on the website in 2024.

As part of NeurologyLive®'s Year in Review, take a look at our most-read news in neuromuscular disorders in 2024.

As part of NeurologyLive®'s Year in Review, we've compiled the top coverage stories from 2024 across a variety of different neurological conferences the team either attended or covered virtually.

As part of NeurologyLive®'s Year in Review, we've compiled the most-read feature-length stories that appeared on the website in 2024.

The complement system is critical in immune defense and tissue homeostasis, but its dysregulation can contribute to autoimmune neurological disorders and neurodegenerative diseases like Alzheimer, ALS, and multiple sclerosis.

Vatiquinone, a small molecule inhibitor, demonstrated its efficacy and safety across a range of age groups with Friedreich ataxia, with effects seen on disease progression and specific subscales of the mFARS.

Human studies revealed thinner myelin sheaths in CD59-deficient patients, indicative of a process of segmental demyelination followed by remyelination.

Neurology News Network. for the week ending December 21, 2024. [WATCH TIME: 4 minutes]

Managing ALS, particularly its respiratory complications, requires a collaborative multidisciplinary team—including neurologists, pulmonologists, and respiratory therapists—working together to address the complexities of respiratory failure, the leading cause of death in these patients.

The program assembles a collaborative team of in-house and external experts in research, clinical care, and regulatory affairs to navigate gene therapy programs through the drug development process.