Marco Meglio

Roche has updated its phase 2 GENERATION HD2 trial to continue testing only the higher 100 mg dose of tominersen, following interim data favoring its potential clinical benefit in Huntington disease.

NeuroOne's OneRF system, already cleared for functional neurosurgery, is now being evaluated by the FDA for trigeminal nerve ablation to treat chronic facial pain.

The findings support the neuroprotective effects of GLP-1RAs and SGLT2is, suggesting their possible role in Alzheimer disease prevention strategies in people with type 2 diabetes.

In this phase 2 trial, TTNS using the Geko device was safe and well tolerated but showed no significant benefit over sham in improving urinary symptoms in Parkinson disease.

Matthew Wicklund, MD, a professor of neurology at the University of Texas Health Science Center San Antonio, offered a clinical and translational overview of OPMD, highlighting current care challenges and the early promise of dual-action gene therapy strategies.

In the LEARN trial, long-term MMF treatment in NMDARE reduced relapse risk, improved seizure control and symptom burden, and was well tolerated over 24 months.

Sonya Miller, medical director at TauRx, discussed the latest clinical data and development plans for HMTM, a potential oral therapy targeting tau pathology in Alzheimer disease.

A global cohort study identified distinct clinical patterns and higher relapse rates in pediatric patients with MOGAD with non-P42 MOG antibody binding profiles.

Ken Mariash, chief executive officer at Sinaptica, provided commentary on new data from the company’s SinaptiStim neuromodulation system, highlighting its clinical promise in slowing Alzheimer disease progression through targeted, non-drug brain network stimulation.

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 stroke in epilepsy.

Backed by promising early data and mechanistic insights, SNUG01 enters clinical trials as the first TRIM72-based gene therapy candidate for ALS.

Neuromuscular expert Matthew Alexander, PhD, explores the evolving landscape of targeted therapies in muscular dystrophies, from fibrosis and glycosylation to combination strategies with gene therapy.

The former FDA commissioner discussed the collaborative power of the neuromuscular community and the challenges of implementing emerging therapies in a rapidly evolving care landscape. [WATCH TIME: 4 minutes]

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Nancy Foldvary-Schaefer, DO, FAAN. [LISTEN TIME: 25 minutes]

The chief scientific officer at Avidity Biosciences details the mechanism, early results, and future plans for Del-zota, an investigational therapy targeting exon 44 in Duchenne muscular dystrophy.

Using discovery and validation cohorts, plasma miRNA-214 levels were shown to correlate with disease severity and were also associated with plasma neurofilament light and CSF inflammatory cytokines.

Philip Kremer, MD, Research Director Neurology, Center for Human Drug Research, provided clinical insights on the early-stage progress of ARV-102, an LRRK2 degrader, in LRRK2-associated diseases.

Afterimage duration was more prolonged in patients with migraine with aura than those without, suggesting differing underlying pathophysiology between the two subtypes despite the exact mechanisms remaining unclear.

The intranasal treatment, originally marketed in 2020, will now be available for patients with acute repetitive seizures as young as 2 years old.

Beth Stein, MD, director of neuromuscular diseases at St. Joseph’s Health, shared insights on the impact of a newly approved pre-filled syringe formulation of efgartigimod, highlighting its potential to transform care delivery.

The chief scientific officer at Satellos Bioscience discussed phase 1 findings on SAT-3247, an oral AAK1 inhibitor, highlighting its safety, pharmacokinetics, and early translatability in Duchenne muscular dystrophy. [WATCH TIME: 7 minutes]

CT-132, an approach that aims to reduce brain hypersensitivity, becomes the first marketed digital treatment to prevent migraine, intended to be used with other previously approved medications.

In a late-stage trial, telitacicept demonstrated significant and rapid clinical improvements in gMG, achieving notable symptom reductions, improved quality of life, and strong safety outcomes.

High doses of RNS60 led to greater number of patients with modified Rankin scale scores between 0-2, improved EQ-5D-5L index score, and enhanced NIHSS score at each pre-specified time point over placebo.

The vice president of research at Cognition Therapeutics provided commentary on the safety of investigational CT1812 and its role in immune response and synapse-related pathways. [WATCH TIME: 3 minutes]

Elizabeth Head, PhD, a professor in the Department of Pathology and Laboratory Medicine at the University of California, Irvine, shed light on the emerging research and trials surrounding the interplay between Alzheimer disease and Down syndrome.

Patients treated with efgartigimod for myasthenia gravis were found to have a higher incidence and severity of infections compared to those treated with immunoglobulins.

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 essential tremor.

The phase 1 LUMINA trial evaluates Amylyx Pharmaceuticals' AMX0114, an antisense oligonucleotide targeting calpain-2, as a potential therapy for ALS by focusing on safety, tolerability, and biomarkers tied to disease progression.

ARV-102 showed dose-dependent LRRK2 degradation and favorable safety in its first-in-human trial, supporting its promise for treating LRRK2-linked neurodegenerative diseases.