Neuromuscular

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.

Zevra Therapeutics plans to immediately start its launch activities for arimoclomol, marketed as Miplyffa, which is anticipated to be commercially available in 8 to 12 weeks in the United States.

The phase 1b/2a dose escalation trial of KINE-101, conducted in Korea, includes patients with chronic inflammatory demyelinating polyneuropathy who have received at least 1 frontline treatment regimen.

Neal K. Shah, CEO of CareYaya Health Technologies, discussed a miniaturized brain-machine interface developed that could restore communication for patients with neurological disorders by translating brain activity into text.

In the study, patients with spinocerebellar ataxia treated with troriluzole demonstrated statistically significant improvements at years 1 and 2 on the treatment.

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

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

Known as QRL-101-03, the phase 1 study is expected to include 60 healthy participants, with topline results expected in the first half of 2025.

A pair of neurologists from the University of California, Irvine, provided commentary on the ways to help young investigators find their footing in research settings, and exposing them to opportunities major governmental agencies can provide. [WATCH TIME: 4 minutes]

A 28-year-old patient initially diagnosed with Guillain–Barré Syndrome presented with multiple cranial neuropathies and was later rediagnosed with chronic inflammatory demyelinating polyneuropathy.

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

The general neurologist at Brigham and Women’s Hospital described the approach and mindset in teaching neurology as the field continuously evolves. [WATCH TIME: 3 minutes]