Neuromuscular

The U.S. HHS announced 15 drugs, including deutetrabenazine, will undergo Medicare Part D price negotiations to lower costs for seniors by 2027.

Previously, Argenx's efgartigimod alfa and hyaluronidase-qvfc was granted orphan drug designation for the treatment of chronic inflammatory demyelinating polyneuropathy in Japan.

Crossover-treated patients demonstrated clinically meaningful and statistically significant improvements in functional outcomes despite being older at the time of treatment.

Neurology News Network. for the week ending January 25, 2025. [WATCH TIME: 4 minutes]

In the phase 2/3 DEVOTE study, a higher dose of nusinersen met its primary end point at 6 months, achieving a statistically significant improvement in motor function among treatment-naïve symptomatic infants with SMA.

Take a look at some of the most-anticipated FDA pending approvals expected in 2025 that researchers and clinicians in neurology should keep an eye out on.

The study highlighted tofersen's potential to slow ALS progression in patients with SOD1 mutations, showing reduced neurofilament levels, functional stabilization, and improved muscle strength, despite mild to moderate adverse events.

Robert Rothrock, MD, a spinal neurosurgeon at Baptist Health Miami Neuroscience Institute, provided commentary on key topics in spine health amid the 2025 Baptist Health Spine Symposium.

The professor and chair of the Department of Molecular Pathobiology at NYU College of Dentistry discussed how a recently published preclinical study adds to the understanding of neuropilin-1 in chronic pain. [WATCH TIME: 3 minutes]

A new study highlighted the importance of initiating immunotherapy promptly in chronic inflammatory demyelinating polyneuropathy to prevent axonal damage and disability progression.

A study highlighted corneal confocal microscopy as a promising noninvasive tool for tracking sensory nerve damage in chronic inflammatory demyelinating polyradiculoneuropathy.

Nipocalimab’s biologics license application is supported by the phase 3 Vivacity-MG3 trial, in which the agent demonstrated superiority over placebo plus standard-of-care over a 24-week period.

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]

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Russell Lebovitz, MD, PhD. [LISTEN TIME: 22 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.