Neuromuscular

Managing ALS, particularly its respiratory complications, requires a collaborative multidisciplinary team—including neurologists, pulmonologists, and respiratory therapists—working together to address the complexities of respiratory failure, the leading cause of death in these patients.

The program assembles a collaborative team of in-house and external experts in research, clinical care, and regulatory affairs to navigate gene therapy programs through the drug development process.

The proof-of-mechanism study will evaluate single doses of QRL-101 in approximately 12 patients with ALS, focusing on excitability biomarkers like the strength-duration time constant (SDTC), safety, tolerability, and plasma pharmacokinetics, with topline results expected in early 2025.

As of July 2024, 2 participants in the ongoing CONNECT1 study have received 4 doses of PGN-EDO51 at 10 mg/kg, which has been generally well tolerated, with initial results expected in early 2025.

The positive data, along with data from GRAND CANYON anticipated in early 2025, are thought to be part of a potential future authorization submission for sevasemten in Becker.

Donald S. Wood, PhD, president and chief executive officer at the MDA, provided thoughts and perspectives on the promising design and educational attainment for the 2025 MDA Clinical & Scientific conference.

The FDA recommended a follow-up meeting with Clene to further discuss the analyses needed to support the accelerated approval pathway for CNM-Au8 in amyotrophic lateral sclerosis.

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

RGX-202 demonstrated improved functional outcomes and robust microdystrophin expression, with levels exceeding those of other approved or investigational gene therapies.

Perspective from neurologists on the current and future applications for artificial intelligence across a bevy of neurologic conditions.

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.

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 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.

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.