137: Advancing Neuromuscular Care and Research

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, "Down Syndrome and Alzheimer’s: Clinical Trials, Equity, and Patient-Centered Progress," Elizabeth Head, PhD, professor in the department of Pathology and Laboratory Medicine at the University of California, Irvine, discussed the evolving intersection of Down syndrome and Alzheimer disease using updates from the Alzheimer Biomarker Consortium – Down Syndrome (ABC-DS). She detailed how outcome assessments have been refined to meet the unique cognitive profiles of individuals with Down syndrome and shared the progress in launching clinical trials that now include this historically excluded population. Head also addressed the challenges in distinguishing developmental delay from early dementia symptoms, the potential role of anti-amyloid therapies, and how non-pharmacological interventions like sleep apnea treatment may contribute to cognitive preservation. She emphasized the importance of equitable research participation, clinician education, and understanding patient priorities—like independence and respect—when supporting individuals with Down syndrome and their families. Looking for more Alzheimer disease/dementia discussion? Check out the NeurologyLive® Alzheimer disease/dementia clinical focus page. Episode Breakdown: 1:00 – Overview of the ABC-DS initiative and the growing inclusion of Down syndrome in Alzheimer research 2:00– Current state of clinical trials and progress in therapeutic development for this population 3:30 – Evolution of outcome assessments tailored for individuals with Down syndrome 5:40 – Challenges in distinguishing Down syndrome traits from signs of early dementia 7:40 – Clinician educations needs and improving access to specialized care 9:00 – Neurology News Minute 11:00 – Eligibility and considerations for anti-amyloid therapies in Down syndrome 13:35 – Research gaps, co-occurring conditions, and the importance of reaching underserved populations 16:40 – Top priorities from patients and families: independence, inclusion, and respect in care settings 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: GENERATION HD2 Trial of Huntington Agent Tominersen Amended to Include Only Higher Dose FDA Grants Authorization to Epiminder’s Implantable Continuous EEG Monitor for Epilepsy Treatment FDA Approves FcRn Blocker Nipocalimab for Broad Forms of Generalized Myasthenia Gravis 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, "Sleep and Seizures: Emerging Insights From AAN 2025," Nancy Foldvary-Schaefer, DO, director of the Sleep Disorders Center at Cleveland Clinic, reflected on key sleep-related topics presented at this year’s AAN Annual Meeting. She highlighted the growing clinical interest in underrecognized conditions like idiopathic hypersomnia and narcolepsy, stressing the need for better awareness and earlier diagnosis. Foldvary-Schaefer also detailed the development of the Sleep by Cleveland Clinic mobile app, designed to improve screening for common sleep disorders with validated tools and real-time resources. Additionally, she discussed new findings on the prevalence of obstructive sleep apnea in adults with epilepsy, pointing to the importance of routine sleep screening in this population, regardless of seizure severity. Finally, she shared exploratory data on SSRI use and peri-ictal respiratory patterns in epilepsy, offering new perspectives on potential protective mechanisms tied to SUDEP risk. Looking for more neuromuscular discussion? Check out the NeurologyLive® Sleep disorders clinical focus page. Episode Breakdown: 1:00 – Key sleep topics discussed at AAN 2025 and the growing interest in hypersomnia 5:45 – Development and purpose of the SLEEP mobile app by Cleveland Clinic 10:35 – Findings on obstructive sleep apnea prevalence in adults with epilepsy 15:05 – Neurology News Minute 17:10 – The need for sleep disorder screening in epilepsy clinical trials 19:45 – Exploratory findings on SSRI use and respiratory patterns in patients with epilepsy 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 Pre-Filled Syringe Administration for FcRn Modulator Efgartigimod FDA Approves CT-132 as First Digital Therapeutic for Preventive Treatment of Episodic Migraine FDA Expands Diazepam Nasal Spray Indication to Treat Ages 2 to 5 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, "Training the Next Generation of Neurology Nurse Practitioners," Daniel Harrison, MD, neurointensivist and assistant professor of neurology at Boston Medical Center, discussed his role as a senior author on a paper focused on a national needs assessment survey of neurologic education in nurse practitioner (NP) programs. In the conversation, Harrison explained how the survey aimed to understand neurology education across NP programs and identify gaps in training for advanced practice providers. He highlighted key findings, including the importance of lesion localization and neuroradiology for NP postgraduate training. While basic neurology education is integrated into many NP programs, he emphasized the need for more hands-on clinical experiences in specialized areas like neurocritical care and neuro-oncology. He also discussed the critical shortage of preceptors for neurology rotations, which hinders clinical education. Looking ahead, Harrison shared his thoughts on the growing role of NPs in neurology, forecasting a substantial increase in their numbers over the next decade and its impact on both clinical care and education. He also acknowledged the contributions of his colleague, Kate Sweeter, NP, who played a key role in the research, highlighting the importance of NPs in clinical care and academic advancements in neurology. Looking for more neuromuscular discussion? Check out the NeurologyLive® Neuromuscular clinical focus page. Episode Breakdown: 1:00 – Overview of the national needs assessment survey  3:45 – Importance of lesion localization and neuroradiology for NP postgraduate training 6:05 – The role of neuropathology in neurology education for nurse practitioners 8:05 – Challenges with clinical rotation preceptors and the shortage in neurology 9:40 – Neurology News Minute 12:10 – The role of NP programs in providing clinical experience 14:55 – Variability and lack of standardization in NP neurology education 18:05 – Evolving duties and responsibilities of NPs over the coming years 19:35 – Closing thoughts, acknowledging the contributions of Kate Sweeter, NP, and the value of NPs in neurology 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 Accepts Regulatory Submission for BTK Inhibitor Tolebrutinib for Non-Relapsing Secondary Progressive MS FDA Approves Diazoxide Choline Extended-Release Tablets for Hyperphagia in Prader-Willi Syndrome Genentech Reports High-Dose Ocrelizumab Fails to Show Additional Benefit in Relapsing MS 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 this special episode of the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. For major FDA decisions in the field of neurology, we release short special episodes to offer a snapshot of the news, including the main takeaways for the clinical community, as well as highlights of the efficacy and safety profile of the agent in question. In this episode, we cover the recent approval of Supernus Pharmaceuticals' agent SPN-830, marketed as Onapgo, as the first and only subcutaneous apomorphine infusion device for the treatment of motor fluctuations in adults with advanced Parkinson disease (PD). SPN-830, which has been available in Europe for several years, was approved based on data from the TOLEDO study (NCT02006121), a randomized, double-blind study in which treatment with the device was associated with a difference of –1.89 hours per day of OFF time for patients with PD in comparison with placebo. To gain greater insights on how this approval impacts the clinical and patient communities, NeurologyLive® sat down with movement disorder experts Stuart Isaacson, MD, director of the Parkinson’s disease and movement disorders center of Boca Raton, and Rajesh Pahwa, MD, the Laverne and Joyce Rider professor of neurology at the University of Kansas Medical Center. The duo discussed the significance of the approval, considerations for patient selection with the treatment, as well as the studies supporting its safety and efficacy. In addition, the pair provided context on how this approval, along with other recent approvals in PD, speak to the progress the clinical and research community has made over the years. For more of NeurologyLive's coverage of SPN-830 for Parkinson disease, head here: FDA Approves Apomorphine Infusion Device SPN-830 as New Parkinson Treatment Episode Breakdown: 1:05 – Significance of SPN-830's approval and impact on PD community 3:10 – Considerations for treatment selection with SPN-830, conversations with families 5:50 – Neurology News Minute 7:50 – TOLEDO study, supportive efficacy and safety data for SPN-830 11:45 – Expanding PD treatment options and overviewing recent progress in research 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 Vutrisiran for ATTR-CM, Expanding Indication in Amyloidosis FDA Accepts Regulatory Submission for BTK Inhibitor Tolebrutinib for Non-Relapsing Secondary Progressive MS Simufilam Fails to Meet Key End Points in Phase 3 Alzheimer Disease Trial 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, "Advancing Neuromuscular Care and Research," former FDA commissioner Robert Califf, MD, explored the urgent need for sustained NIH funding to advance basic and translational research, including gene editing, assistive devices, and the integration of artificial intelligence. He emphasized the collaborative spirit of the neuromuscular community, where shared technologies and insights can address multiple disorders. The conversation, which took place at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific conference, highlighted critical challenges, such as clinician shortages, healthcare system strain, and the need for post-market learning to manage uncertainties with emerging therapies like gene therapy. Additionally, he called for innovative funding solutions to address the high costs of treatment, ensure equitable access, and maintain progress in neuromuscular care while fostering long-term health equity. Looking for more epilepsy discussion? Check out the NeurologyLive® neuromuscular clinical focus page. Episode Breakdown: 1:05 – Funding priorities for neuromuscular research and NIH support 1:55 – Role of gene editing, assistive devices, and AI in care 4:50 – Collaboration and togetherness of the neuromuscular community 6:35 – Considerations as new therapeutics emerge for neuromuscular disorders 8:05 – Funding solutions for equitable care 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 this special episode of the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. For major FDA decisions in the field of neurology, we release short special episodes to offer a snapshot of the news, including the main takeaways for the clinical community, as well as highlights of the efficacy and safety profile of the agent in question. In this episode, we cover the recent approval of Genentech's tenecteplase, marketed as TNKase, as a new treatment for adults with acute ischemic stroke (AIS). The thrombolytic medicine is an intravenous tissue plasminogen activator that is clot-dissolving, administered as a single 5-second intravenous bolus. Tenecteplase is only the second such approval for this indication, following the 2015 approval of alteplase (Activase; Genentech); however, tenecteplase is considered faster and more simply administered than alteplase. To better understand the implications of this approval, NeurologyLive sat down with stroke expert Bijoy Menon, MD, MSc, FRCPC, a professor of neurology at the University of Calgary. In the interview, Menon talked about the downstream impacts of having another approved therapy, how tenecteplase differs from other thrombolytic agents, and the efficacy and safety that supported its approval. Furthermore, he provided commentary on how the approval continues to chip away toward the idea of precision medicine and personalizing treatments for patients with AIS.  For more of NeurologyLive's coverage of sodium oxybate's (Lumryz) expanded indication, head here: FDA Approves Tenecteplase for Acute Ischemic Stroke Episode Breakdown: 1:40 – Immediate reaction and significance of tenecteplase approval 5:30 – Safety considerations and administration when prescribing tenecteplase 10:20 – Supportive efficacy and safety, phase 3 AcT trial, and other supplementary studies 14:50 – Advancing precision medicine, treatment personalization with new approval 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, Sonal Bhatia, MD, FACNS, epilepsy expert and director of the pediatric epilepsy monitoring unit (EMU) at the Medical University of South Carolina, discussed Purple Day, celebrated on March 25th, and the importance of raising awareness for patients with epilepsy, a common condition affecting one in 26 individuals. She discussed the stigma faced by patients with epilepsy, especially in parts of the world outside the United States. Bhatia also discussed some of the recent advances in epilepsy care in the past decade, highlighting areas like improved surgical approaches, dietary therapies, and the potential of gene therapy for various disorders. In addition, she discussed changes in elderly epilepsy care, as well as the importance of transitioning care from pediatrics to adults and the need for a longitudinal model of care. Furthermore, she spoke on epilepsy monitoring units, the challenges institutions face, and the critical need for EEG technology and for more EEG technologists and nursing school graduates.  Looking for more epilepsy discussion? Check out the NeurologyLive® Epilepsy clinical focus page. Episode Breakdown: 1:10 – Significance of Purple Day, raising awareness on epilepsy stigma 5:00 – Advances in epilepsy care, including availability of more anti-seizure medications and genetic insights 8:45 – Neurology News Minute 11:10 – Challenges in elderly epilepsy care and the need for more standardized transition models from pediatrics to adulthood 15:20 – Optimizing epilepsy monitoring units and dealing with staff and equipment shortages 20:50 – Closing thoughts 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 Medtronic’s Adaptive Deep Brain Stimulation for Parkinson Disease FDA Approves Tenecteplase for Acute Ischemic Stroke FDA Approves Expanded Use of Eculizumab for Pediatric Myasthenia Gravis 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, Julie Flygare, JD, president and chief executive officer of Project Sleep, shared her personal and professional insights into the critical role of social connections for patients living with narcolepsy and idiopathic hypersomnia. Drawing from her journey with narcolepsy, Flygare highlighted the challenges of isolation at diagnosis and the transformative power of peer support and advocacy. She delved into survey findings that underscore the rarity of social connections among patients and their impact on adjustment and well-being. Additionally, Flygare offered actionable recommendations for clinicians to integrate social support into practice, emphasizing the value of patient advocacy organizations and fostering meaningful connections. Above all, her inspiring perspective shed light on the intersection of community, research, and clinical care in sleep health. Looking for more narcolepsy discussion? Check out the NeurologyLive® Narcolepsy clinical focus page. Episode Breakdown: 1:00 – Challenges and importance of social connections 5:45 – Building a thriving community and research impact 7:30 – Insights from patient surveys on social support 10:30 – Neurology News Minute 12:25 – Recommendations for clinicians on social support 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 Mirdametinib for NF1-Associated Plexiform Neurofibromas in Adults and Children FDA Approves Tablet Formulation of Risdiplam for Spinal Muscular Atrophy Solid Reports Positive Data for SGT-003 Gene Therapy in Phase 1/2 Trial of Duchenne 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, Veronique Miron, PhD, The John David Eaton Chair in Multiple Sclerosis at the University of Toronto, gave a clinical overview of the 2025 ACTRIMS Forum, which is held February 27-March 1, in West Palm Beach, Florida. Miron, an expert in the field, spoke on the reasons behind this year's theme of "Making Connections," as well as some of the most notable and innovative sessions attending clinicians should keep their eyes on. Additionally, she spoke on how the curriculum at this year's meeting aligns with the most recent MS McDonald Diagnostic Criteria that was presented at ECTRIMS 2024. Miron also spoke on the different seminars that cover drug development, innovative new treatment approaches, and Bruton Tyrosine Kinase (BTK) inhibitors. Furthermore, she provided commentary on how this year's meeting differs from previous iterations of ACTRIMS, highlighting some of the changes to the agenda and the advances in MS research. Looking for more multiple sclerosis discussion? Check out the NeurologyLive® Multiple sclerosis clinical focus page. Episode Breakdown: 1:10 – Reasons behind "Making Connections" theme  2:50 – Overviewing key unique sessions and forums from the meeting 5:10 – Aligning curriculum with 2024 MS McDonald Criteria 7:00 – Neurology News Minute 9:00 – Drug development topics from ACTRIMS 2025 12:20 – Differences between ACTRIMS 2025 vs ACTRIMS 2024 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 Axsome Therapeutics’ AXS-07 for Migraine Treatment FDA Approves Vertex Pharmaceuticals' Suzetrigine for Acute Pain Management FDA Approves Apomorphine Infusion Device SPN-830 as New Parkinson Treatment 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, epilepsy expert Depobam Samanta, MD, MS, FAAP, FAES, sat down to discuss a recently published guidance paper from members of the Pediatric Epilepsy Research Consortium that provides recommendations for neuromodulation approaches to treat Lennox-Gastaut syndrome (LGS). Samanta, medical director of the Arkansas Children's Comprehensive Epilepsy Program, gave a clinical overview of the paper, highlighting the various neuromodulatory devices and their use, and the ways to improve treatment selection and personalization. Additionally, he talked about some of the potential complications with these devices as well as initiation and titration strategies for easy initiation. Furthermore, he spoke about ways to take neuromodulation to the next level, giving insight on creative trial designs, overcoming sham-controlled studies, and the necessary research to expand these approaches.  Looking for more epilepsy discussion? Check out the NeurologyLive® Epilepsy clinical focus page. Episode Breakdown: 1:05 – Overview of the published guidance and reasons behind the paper  5:15 – Patient selection for neuromodulation and tailoring treatments by patient and preference 8:25 – Overcoming complications with neuromodulation devices  11:25 – Multidisciplinary team necessary for successful implementation of neuromodulation 13:15 – Neurology News Minute 15:15 – Initiation and titration strategies for vagus nerve stimulation, deep brain stimulation, and resective surgery 18:30 – Next steps in research and expanding neuromodulation for patients with LGS 21:10 – Finding creative trial designs to test neuromodulation devices, combination approaches 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 Accepts BLA for Subcutaneous Autoinjector Formulation of Lecanemab Cell Therapy Bemdaneprocel Advances to Phases 3 Registrational Trial in Parkinson Disease Real-World Study Highlights Positive Treatment Benefits of Tofersen on ALS Disease Progression, Function 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.