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.

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.

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.

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]

Investigators are set to test the effects of 300 mg/day fasudil after the therapy showed promising data on ALS-related biomarkers in 180 mg/day doses.

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

Test your neurology knowledge with NeurologyLive®'s weekly quiz series, featuring questions on a variety of clinical and historical neurology topics. This week's topic is on Friedreich ataxia.

A safety analysis of the phase 3 MOXIe Part 2 trial (NCT0225435) highlighted the frequency and timing of TEAEs with omaveloxolone (Skyclarys; Biogen) treatment.

Neurology News Network. for the week ending November 9, 2024. [WATCH TIME: 4 minutes]

NGN-401, an AAV9 gene therapy in development for Rett syndrome, was considered well-tolerated, with treated patients showing consistent gains in skill and developmental milestones.

The senior scientist at Sunnybrook Research Institute in Toronto, Ontario, provided clinical insight on the advantages of a flipped classroom, and why neurology educators should consider this approach. [WATCH TIME: 2 minutes]

Italo Linfante, MD, director of interventional neuroradiology at Baptist Health Miami Neuroscience Institute, discussed the importance of brain aneurysms in the context of neurology.

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