NeurologyLive® Mind Moments®

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice.In this Mind Moments episode, M. Scott Perry, MD, head of neurosciences at Cook Children's Medical Center, discussed how epilepsy trial design is evolving, including basket trials that group related conditions, approaches to reduce placebo exposure, and greater inclusion of children under the age of 2 years. He also highlighted a growing focus on outcomes beyond seizure frequency, such as cognition, development, and behavior, particularly in developmental epileptic encephalopathies. During the conversation, Perry also described electroencephalography (EEG) measures as an emerging exploratory biomarker and sees potential for genetic, serum, and imaging markers to help identify patients most likely to benefit from treatment. Looking ahead, Perry noted that he envisions more targeted trials that may be smaller and shorter, with less reliance on placebo as researchers better understand disease biology and natural history. Looking for more Epilepsy discussion? Check out the NeurologyLive® Epilepsy focus page.Episode Breakdown: 1:10 - Evolution of epilepsy trial design  3:44 - Outcomes beyond seizure frequency  5:38 - Biomarkers in current trials  6:17 - Neurology News Minute   8:48 - Biomarkers and epilepsy heterogeneity  10:35 - Genetic screening in epilepsy trials  12:38 - Vision for future clinical trials  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: FDA Approves Autoinjector for Self-Administered Zilucoplan in Generalized Myasthenia Gravis FDA Grants Fast Track to AV-1959R for Alzheimer Prevention FDA Accepts Troriluzole NDA Resubmission for Spinocerebellar Ataxia Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice.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, director of the ALS Clinic and director of clinical trials at NYU Langone Health, join the podcast to preview the 2026 Network of Excellence for ALS (NEALS) Annual Meeting, taking place October 27-30 in Clearwater, Florida. The duo discusses the meeting's major scientific themes, emerging clinical research, and the growing emphasis on earlier intervention, precision medicine, and innovative therapeutic development for amyotrophic lateral sclerosis (ALS).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.Looking for more Neuromuscular discussion? Check out the NeurologyLive® Neuromuscular clinical focus page.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 20:40 – Key clinical takeaways emphasizing scientific collaboration, patient involvement, and future ALS treatment development 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 Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice.In this special episode of Mind Moments, released in recognition of Limb-Girdle Muscular Dystrophy (LGMD) Awareness Day on September 30, Katherine Mathews, MD, professor of pediatrics and neurology at the University of Iowa Carver College of Medicine, joins the podcast to discuss the evolving treatment landscape for LGMD and the potential significance of BBP-418, an investigational oral therapy for LGMD2I/R9.With an FDA decision expected by November 27, 2026, BBP-418 could become the first approved therapy for LGMD2I/R9 and potentially the first treatment approved for any form of LGMD. Mathews breaks down the therapy's FKRP-targeted mechanism, findings from the phase 3 FORTIFY study, and the challenges of conducting clinical trials across genetically diverse and slowly progressive forms of LGMD. She also discusses what a first approval could mean for the broader LGMD community, considerations for bringing patients back into specialty care, and research priorities that could accelerate development of future therapies.Looking for more Neuromuscular discussion? Check out the NeurologyLive® Neuromuscular clinical focus page.Episode Breakdown: 1:15 – BBP-418's mechanism and therapeutic rationale for FKRP-related LGMD2I/R9 4:00 – FORTIFY findings supporting BBP-418's potential effects on muscle and pulmonary function 7:50 – Why genetic heterogeneity and slow progression complicate LGMD clinical trials 10:10 – Neurology News Minute 12:45– What a first FDA-approved LGMD therapy could mean for patients and clinicians 15:40 – Research priorities for accelerating clinical trials and developing future LGMD therapies 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: CHMP Recommends EU Approval for Ocrelizumab in Pediatric Relapsing MS SKY-0515 Shows Sustained Benefit at 15 Months in Huntington Disease FDA Approves Tavapadon for the Treatment of Parkinson Disease Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice.In this Mind Moments episode, Jennifer Fokas, MD, and Minali Nigam, MD, neurologists at Northwestern Medicine with specialized training in headache medicine, join the podcast to discuss current challenges and evolving strategies in the management of cluster headache. They highlighted the substantial burden of disease, particularly among patients with chronic cluster headache, and the diagnostic difficulties that arise from overlap with migraine features. They emphasized the importance of recognizing cranial autonomic symptoms as key distinguishing features, along with typical attack duration and circadian or seasonal patterns. The conversation also covered practical approaches to acute and preventive treatment, including high-flow oxygen via non-rebreather mask, subcutaneous sumatriptan, bridge therapies, and preventive options such as high-dose verapamil and, in selected cases, lithium. The physicians also review emerging and adjunctive options while noting limitations in evidence, indications, and access. They concluded by underscoring the need for clinician preparedness and tailored therapy to enable more assertive and appropriate management of cluster headache. Looking for more Headache and Migraine discussion? Check out the NeurologyLive® Headache and Migraine focus page.Episode Breakdown: 1:54 – Biggest unmet need in cluster headache 5:01 – Importance of multimodal acute management and access issues 7:39 – Neurology News Minute  10:03 – Emerging and experimental therapies 13:03– Preventive options and special situations 17:51 – Key clinical takeaway: preparation and access to therapies 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: FDA Approves Zilganersen, First Treatment for Alexander Disease FDA Priority Review Puts Satralizumab on Track as First MOGAD Drug FDA Approves Apitegromab for Spinal Muscular Atrophy Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.REFERENCE1. Burish MJ, Pearson SM, Shapiro RE, Zhang W, Schor LI. Cluster headache is one of the most intensely painful human conditions: Results from the International Cluster Headache Questionnaire. Headache. 2021;61(1):117-124. doi:10.1111/head.14021

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice.In this Mind Moments episode, Aaron Zelikovich, MD, a neuromuscular physician at Lenox Hill Hospital, part of Northwell Health, joins the podcast to discuss the emerging conversation around dual therapy in spinal muscular atrophy (SMA). As disease-modifying treatments continue to reshape survival and long-term outcomes, Zelikovich examines whether combining SMN-targeted approaches could provide additional benefit, while emphasizing that evidence supporting dual therapy remains limited and significant barriers to studying it persist. The conversation explores clinician and patient interest in combination treatment, insurance and access challenges, and what current evidence suggests about adding therapy after gene replacement, particularly in children who are not meeting expected motor milestones. Zelikovich also discusses the need for better biomarkers to guide treatment decisions, the changing natural history of adult SMA, and why stronger real-world data and prospective research will be critical to determining whether dual therapy ultimately has a place in SMA care. Looking for more Neuromuscular discussion? Check out the NeurologyLive® Neuromuscular clinical focus page.Episode Breakdown: 1:05 – Clinical perspectives and current controversy surrounding dual therapy in SMA 2:40 – Discussing dual therapy expectations and uncertainty with patients and families 4:35 – Evidence guiding when clinicians should consider adding a second therapy 6:20 – Neurology News Minute  8:55– Developing better biomarkers to guide long-term SMA treatment decisions 11:15 – Research needed to evaluate dual therapy and adult SMA outcomes 13:35 – Moving the dual therapy conversation forward through patient-clinician partnerships 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: FDA Clears Roche's Single-Biomarker Elecsys pTau217 Blood Test for Alzheimer Disease FDA Extends Deramiocel Review for Duchenne Muscular Dystrophy After HOPE-3 Amendment AHA/ASA Issue New Guideline for Adult Stroke Rehabilitation and Recovery Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.