Three-year extension data from the phase 3 IB1001-301 trial show levacetylleucine slowed disease progression in both adults and children with Niemann-Pick disease type C.
Commentary|Podcasts|October 16, 2026
Episode 177: Previewing the 2026 NEALS Annual Meeting
Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Nicholas J. Maragakis, MD; and Jinsy Andrews, MD, MSc, FAAN, FANA. [LISTEN TIME: 23 minutes]
Advertisement
Episode 177 of the NeurologyLive®
The Mind Moments podcast features exclusive interviews with leaders in the field discussing the latest research and disease management strategies across the breadth of neurology, including epilepsy, multiple sclerosis (MS), Parkinson disease, dementia, sleep disorders, and more.
In this episode of Mind Moments, Nicholas J. Maragakis, MD, director of the Center for ALS Specialty Care at Johns Hopkins, and Jinsy Andrews, MD, FAAN, FANA, director of the ALS Clinic and director of clinical trials at
The conversation explores new programming focused on presymptomatic ALS, including the ATLAS and Prevent ALS studies, along with updates on ongoing clinical trials and emerging approaches targeting SOD1, FUS, TDP-43, and other disease-related pathways. Maragakis and Andrews also examine advances in trial design, biomarker-guided treatment strategies, antisense oligonucleotides, and gene-targeted therapies, while highlighting the importance of collaboration among researchers, clinicians, industry partners, and individuals living with ALS. Together, they outline what attendees can expect from NEALS 2026 and how these developments could shape the future of ALS research and clinical care.
The stories featured in this week’s Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here:
Congress Passes ACT for ALS Reauthorization, Extending Research Programs Phase 4 THRIVE Data Highlights Eptinezumab’s Impact on Migraine After Prior CGRP Failure Solengepras Meets Primary Endpoint in Phase 3 ARISE Trial of Parkinson Disease
EPISODE BREAKDOWN
- 1:15 – Overview of NEALS, its expanded clinical research network, and the 2026 meeting's objectives
- 3:00 – New meeting programming focused on presymptomatic ALS, genetic risk, and prevention studies
- 7:05 – Notable clinical trials, emerging therapeutic targets, and research innovations featured at NEALS 2026
- 9:55 – Evolution of ALS clinical trial design, including platform studies, precision medicine, and biomarkers
- 14:10 – Neurology News Minute
- 16:25 – Emerging gene-targeted therapies, ASOs, viral vectors, and lessons from other neuromuscular disorders
- 20:44 – Key clinical takeaways emphasizing scientific collaboration, patient involvement, and future ALS treatment development
Hoping to listen on your favorite podcast app? See below:
Advertisement
Related to this article
Adults with generalized myasthenia gravis who are anti-acetylcholine receptor antibody positive now have a second self-administration option for the complement inhibitor zilucoplan.
The phase 1 ASCEND trial is testing a single intrathecal dose of the investigational AAV9 gene therapy INS1201 in young boys with Duchenne muscular dystrophy.
The designation follows phase 2 ClearMEMory data in which the oral NMDA receptor antagonist improved objective cognitive performance over placebo at 12 weeks.
Arimoclomol Plus Miglustat Shows Early, Sustained Slowing of Niemann-Pick Disease Type C Progression
A post hoc analysis of the phase 2/3 NPC002 trial presented at CNS 2026 showed that the benefit of arimoclomol plus miglustat in Niemann-Pick disease type C emerged as early as 3 months after treatment initiation.
The professor of neurology and program director of Neuromuscular Medicine at UC Irvine, discusses phase 3 NIMBLE trial findings for cemdisiran in generalized myasthenia gravis, including its novel siRNA mechanism, efficacy, and hospitalization data.
New 48-week phase 3 data show many patients with generalized myasthenia gravis reached minimal symptom expression on telitacicept, and most who did sustained it.
New analyses found differences in functional outcomes over 2 years among patients aged 8 to 12 years treated with delandistrogene moxeparvovec, while separate findings characterized micro-dystrophin expression in children younger than 3 years.
Advertisement
Advertisement
Trending on NeurologyLive - Clinical Neurology News and Neurology Expert Insights
1
New Phase 1 Study Tests Intrathecal Gene Therapy Agent INS1201 in Duchenne Muscular Dystrophy
2
MDS Congress 2026: Late-Breaking Data Snapshot
3
Satralizumab Reduces MOGAD Relapse Risk in Phase 3 METEOROID Trial
4
The Road Ahead for Precision Medicine in Multiple Sclerosis
5