Neuromuscular

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending October 11, 2024.

In this discussion, experts provide key considerations in the process of newborn screening for SMA, noting the need for developing local protocols and understanding the limitations of screening infants early in life.

AOC 1001, also known as del-desiran, has shown promising results in improving muscle strength and reducing myotonia in patients with DM1, with patients even reporting better daily activities like hand grip strength.

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

In this episode, experts discuss groundbreaking updates in spinal muscular atrophy diagnosis, emphasizing the importance of early intervention and newborn screening.

After 52 weeks of treatment, no significant differences were found in cardiac MRI measures such as ejection fraction, end diastolic volume, or circumferential strain between the two groups.

The decision follows positive data from multiple trials, showing significant improvement in both cardiac function and muscle performance in patients with DMD.

Alexander disease, a rare leukodystrophy estimated to occur in 1 in 1 million patients in the United States, has no FDA-approved treatments to date.

In this episode, our panel of experts delves into the latest updates on spinal muscular atrophy diagnosis, focusing on the critical role of newborn screening, early intervention, and evolving classification methods for improved patient outcomes.

No serious adverse events were reported, and the therapy demonstrated a favorable safety profile over the 5-year study period.

The hope is that NP001 could potentially slow the progression of the disease through its unique immunologic mechanism.

Overall, vatiquinone-treated patients demonstrated persistent effects on disease progression over a 144-week long-term period, leading to a future regulatory submission.

RAG-18 is an saRNA delivered to muscle tissue to increase UTRN expression and compensate for DMD defects regardless of the location of mutation on DMD gene.

Patients aged 2-21 receiving apitegromab showed clinically meaningful motor function improvements, with a favorable safety profile consistent with long-term data, as Scholar Rock prepares for U.S. and EU regulatory submissions in Q1 2025.

The orphan medicinal product designation given by the European Commission was based on findings produced in research funded by the United States Department of Defense.

Ongoing analysis of vamorolone's effects as a mineralocorticoid receptor antagonist in healthy adults is underway, with results to be presented at upcoming medical conferences.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending October 4, 2024.

Amifampridine enhances neuromuscular transmission and relieves muscle weakness, showing promise for Lambert-Eaton myasthenic syndrome and other neuromuscular junction disorders.

The Total Posterior Spine System offers a promising alternative to spinal fusion, preserving mobility and reducing strain on adjacent spinal levels in patients with spondylolisthesis and spinal stenosis.

As part of our monthly clinician spotlight, NeurologyLive® highlighted neuromuscular-associated cardiomyopathy expert Forum Kamdar, MD, PhD, FACC, FHFSA, an advanced heart failure physician-scientist at the University of Minnesota.

Kelly Brazzo, founder and CEO of CureLGMD2i Foundation, and Dennis Akkaya, chief Commercial Officer at MyTomorrows, share insight on a new partnership that aims to ease accessibility to drug development trials for patients with LGMD.

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

Neurology News Network. for the week ending September 28, 2024. [WATCH TIME: 4 minutes]

Change from baseline in Myasthenia Gravis Activities of Daily Living score was statistically significant for inebilizumab-treated patients and the therapy showed a safe and well-tolerated profile without new safety signals.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending September 27, 2024.

A pair of neurologists from the University of California, Irvine, detailed some of the training and career focuses of large federal agencies in effort to address the shortage of neurologists. [WATCH TIME: 4 minutes]

RNS60 may lengthen survival by slowing FVC progression, particularly in participants with low NfL and MCP-1 levels at baseline, suggesting a potential target subgroup for future studies.

The chief research officer of the Muscular Dystrophy Association talked about what is to be expected at the 2025 MDA Conference, held March 16-19, in Dallas, Texas. [WATCH TIME: 5 minutes]

The company expects to complete the trial in Q1 2025 and anticipates it will use the feedback from regulators to guide an accelerated approval.

The approval follows phase 3 data that showed significant improvements in neurological symptoms and functional benefit over 12 weeks in patients with Niemann-Pick disease type C.