Marco Meglio

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.

Phase 2b trial results showed Descartes-08, a CAR-T therapy, achieved sustained efficacy and a strong safety profile in myasthenia gravis, with notable responses in biologic-naïve patients and no major safety concerns over 12 months.

Cleveland Clinic’s $1.1 billion Neurological Institute, opening in 2027, will revolutionize neurological care with a state-of-the-art, patient-centered design integrating advanced technology and innovative care solutions.

In one of the longest trials of brain stimulation in Alzheimer disease, repetitive transcranial magnetic stimulation led to a reduced decline in CDR-SB, as well as positive impacts on cognitive function and functional abilities.

Emer MacSweeney, MD, a consultant neuroradiologist and trial investigator of the phase 3 APOLLOE4 study, provided a clinical view of the latest study findings, and the difficulties with finding treatments for APOEε4/4 carriers of Alzheimer disease.

The prefilled syringe is approved for a 20- to 30-second subcutaneous injection, and patients can self-inject following proper instruction in the subcutaneous injection technique.

Cassandra Gorsuch, PhD, chief scientific officer at Precision Biosciences, discussed the company’s ARCUS gene editing platform, its innovative approach to treating Duchenne muscular dystrophy, and promising preclinical data presented at MDA 2025.

The professor of neurology at the University of Texas Health Science Center San Antonio discussed some of the challenges with therapeutic development for OPMD, as well as advances and barriers to gene therapy approaches. [WATCH TIME: 4 minutes]

Relative to previously approved therapies like rimegepant, ubrogepant, and zavegepant, AXS-07 resulted in better 2-hour pain relief, sustained pain relief, and reduced use of rescue medications.

Harmony Biosciences’ phase 3 study will assess EPX-100, a repurposed antihistamine, as a potential treatment for Lennox-Gastaut syndrome, targeting seizure reduction and improved outcomes.

Murali Doraiswamy, MD, MBBS, a professor of psychiatry and geriatrics at Duke University School of Medicine, gave clinical follow-up on a pivotal analysis of the phase 3 APOLLOE4 study testing a novel therapeutic for Alzheimer disease.

Denali Therapeutics has begun submitting a BLA for tividenofusp alfa as a potential Hunter syndrome treatment, supported by promising biomarker reductions in clinical trials.