Neuromuscular

A recently initiated phase 1/2 trial of NS-050/NCNP-03 will assess patients with Duchenne muscular dystrophy on dystrophin production, muscle strength, mobility, and functional exercise capacity.

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

Rachana K. Gandhi Mehta, MBBS, an assistant professor of neurology at Wake Forest School of Medicine, discussed a new, innovative trial assessing the therapeutic potential of self-administered subcutaneous rozanolixizumab as a treatment for myasthenia gravis.

QRL-201, developed by QurAlis, aims to restore STATHMIN-2 (STMN2) expression, a key protein for neural repair, which is significantly reduced in patients with ALS.

The vice chair of the American Academy of Sleep Medicine’s Clinical Practice Guidelines Task Force commented on some of the unanswered questions following the recently published guidelines for restless legs syndrome. [WATCH TIME: 3 minutes]

Delaying treatment past 12 months worsens leg disability scores and leads to a more challenging disease course in CIDP patients.

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

Findings from a phase 2/3 study demonstrated the efficacy and safety of a higher dose regimen of nusinersen in treating spinal muscular atrophy in both treatment-naïve and previously treated patients.

Subgroup analyses revealed stronger efficacy signals in biomarker-defined groups, particularly in Caucasian participants with measurable baseline myostatin levels.

These results highlight the age range and ambulation status for which viltolarsen is a key treatment option for DMD patients amenable to exon 53 skipping.

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Andy Berkowski, MD, PhD. [LISTEN TIME: 26 minutes]

Applied Therapeutics noted that it is reviewing the FDA's feedback and will request a meeting to discuss requirements for either a resubmission of the NDA or appeal of the agency's decision.

The professor of neurology at the University of Pennsylvania Perelman School of Medicine provided clinical insight on a long-term safety analysis of omaveloxolone, the first FDA-approved treatment for Friedreich ataxia. [WATCH TIME: 4 minutes]

The vice chair of the American Academy of Sleep Medicine’s Clinical Practice Guidelines Task Force provided in-depth insight on the most noted changes to the 2024 RLS guidelines clinicians should be aware of. [WATCH TIME: 9 minutes]

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

The study, presented at the 2024 AANEM) Annual Meeting, evaluated 52 patients who underwent surgery for carpal tunnel syndrome and explored the comparative effectiveness of the Conway and Zeidman scales in predicting patient outcomes.

Neurology News Network. for the week ending November 23, 2024. [WATCH TIME: 4 minutes]

The multicenter, 24-week study will feature 20 patients with MG, testing changes on MG-ADL as well as several other secondary outcomes, including patient-reported assessments.

Overall, subcutaneous efgartigimod was safe and effective in patients with seropositive MG, demonstrated by consistent improvements in Myasthenia Gravis-Activites of Daily Living.

For more than a 2-year treatment period, 95% of patients reported taking at least 95% of their daily medications with zilucoplan.

The company has decided to pause enrollment in the high-dose cohort of its phase 1 clinical trial for NGN-401 after a participant experienced a serious treatment-related adverse event.

The phase 2 trial is expected to include 80 adults with genetically confirmed CMT who will be followed for a 21-day treatment period, using changes in functional assessments as the primary outcome.

The professor in residence at UCLA Health discussed the critical considerations when deciding appropriate patients for gene therapies, specifically AAV vectors, in treating muscular dystrophies. [WATCH TIME: 4 minutes]

The SKYCLARYS PASS registry, an international observational cohort study, aims to include approximately 300 omaveloxolone-naïve patients participating in the Friedreich’s Ataxia Global Consortium's UNIFIED natural history study.

The professor in residence at UCLA Health provided clinical insight on the limitations of certain gene therapy vehicles for muscular dystrophies, and the need for better vectors that are more myotropic. [WATCH TIME: 3 minutes]