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Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending August 15, 2025. 

NeurologyLive® Friday 5 — August 15, 2025

Videos

The medical director of CNS Clinical Development at AskBio discussed long-term data presented at ATMRD from the company’s phase 1b assessing the investigational gene therapy AB-1005 in Parkinson disease. [WATCH TIME: 6 minutes]  

Overviewing 36-Month, Phase 1b Data Testing Gene Therapy AB-1005 in Parkinson Disease: Nisha Chhabria MD, FAAN

The Harold I. Nemuth Chair in Neurological Disorders at Virginia Commonwealth University discussed the next steps in advancing GLP-1s for idiopathic intracranial hypertension and the research necessary to further understand their therapeutic impact. [WATCH TIME: 3 minutes]

Building a Clinical Trial Framework to Study GLP-1 RAs in Stroke-Related Conditions: Dennis Rivet, MD

A new study reveals rituximab outperforms cladribine in managing MRI disease activity in relapsing multiple sclerosis over 4.5 years.

Target Trial Emulation Favors Rituximab’s Effectiveness Over Cladribine in Treating Multiple Sclerosis

Medical director of CNS Clinical Development at AskBio.

, has been with the team since September 2022. Follow her on Twitter 

At AAIC 2025, Alberto Ramos, MD, FAAN, director of the Sleep Disorders program at the University of Miami, discussed the association between long sleep duration, specific sleep phenotypes, and progressive cognitive decline in Hispanic populations.

Measuring Sleep Duration and Identifying Phenotypes Linked to Cognitive Decline in Hispanic Populations

The clinical assistant professor in movement disorders at Jefferson Health outlined a patient-centered, lifestyle-first approach to managing Parkinson nonmotor symptoms. [WATCH TIME: 4 minutes]

Addressing Constipation, Sleep, and Fatigue in Parkinson Disease Care: Alexa Dessy, MD

Marco Meglio, Assistant Managing Editor for 

, has been with the team since October 2019. Follow him on Twitter 

The associate professor of neurology at Johns Hopkins Medicine previewed a 2025 AUPN panel exploring AI’s clinical, research, and educational applications, along with the ethical considerations shaping its responsible adoption. [WATCH TIME: 2 minutes]

Preparing for the AI-Driven Future of Neurology: Elisabeth Marsh, MD

The neuromuscular condition center is a resource for clinical news on the diagnosis, treatment, and management of neuromuscular conditions. | NeurologyLive.


Neuromuscular | Clinical Neurology News & Insights | NeurologyLive

NeurologyLive is your direct connection to the latest neurology news and interviews with expert neurologists in multimedia formats.

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Clinical

 neuromuscular disease clinical focus page offers updates and information related to the clinical care and management of individuals with neuromuscular diseases such as amyotrophic lateral sclerosis, Duchenne muscular dystrophy, cerebral palsy, and spinal muscular atrophy, as well as rare diseases. This page consists of news, podcasts, and videos about the latest FDA actions, clinical guideline updates, interviews with physicians, study and clinical trial findings, and more.

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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, Parkinson disease, dementia, sleep disorders, and more.

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.

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

 – Overview of the ABC-DS initiative and the growing inclusion of Down syndrome in Alzheimer research

– Current state of clinical trials and progress in therapeutic development for this population

 – Evolution of outcome assessments tailored for individuals with Down syndrome

 – Challenges in distinguishing Down syndrome traits from signs of early dementia

 – Clinician educations needs and improving access to specialized care

 – Eligibility and considerations for anti-amyloid therapies in Down syndrome

 – Research gaps, co-occurring conditions, and the importance of reaching underserved populations

 – Top priorities from patients and families: independence, inclusion, and respect in care settings

 to subscribe to the Mind Moments podcast. Be sure to leave a rating and review for the show. Thanks for listening!

Hoping to listen on your favorite podcast app? See below:

AD/PD International Conference on Alzheimer’s and Parkinson’s Diseases (AD/PD)

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Elizabeth Head, PhD. [LISTEN TIME: 20 minutes]

Episode 140: Down Syndrome and Alzheimer’s: Clinical Trials, Equity, and Patient-Centered Progress

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.

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

 – Overview of the national needs assessment survey 

 – Importance of lesion localization and neuroradiology for NP postgraduate training

 – The role of neuropathology in neurology education for nurse practitioners

 – Challenges with clinical rotation preceptors and the shortage in neurology

 – The role of NP programs in providing clinical experience

 – Variability and lack of standardization in NP neurology education

 – Evolving duties and responsibilities of NPs over the coming years

 – Closing thoughts, acknowledging the contributions of Kate Sweeter, NP, and the value of NPs in neurology

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Daniel Harrison, MD. [LISTEN TIME: 20 minutes]

Episode 138: Training the Next Generation of Neurology Nurse Practitioners

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.

 – Funding priorities for neuromuscular research and NIH support

 – Role of gene editing, assistive devices, and AI in care

 – Collaboration and togetherness of the neuromuscular community

 – Considerations as new therapeutics emerge for neuromuscular disorders

 – Funding solutions for equitable care

Muscular Dystrophy Association Clinical and Scientific Conference (MDA)

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Robert Califf, MD. [LISTEN TIME: 9 minutes]

Episode 137: Advancing Neuromuscular Care and Research

In this episode, "Aiding Diagnosis of Synucleinopathies Through SAAmplify-aSYN Biomarker Test," Russ Lebovitz, MD, PhD, chief executive officer and cofounder of Amprion, discussed the company's SAAmplify-aSYN biomarker test, a first-in-class qualitative laboratory developed test and the only seed amplification assay available to aid in the diagnosis of synucleinopathies such as Parkinson disease (PD) and multiple system atrophy (MSA). Lebovitz provided insight on the new technology and its remarkable accuracy in identifying underling a-synuclein pathology using fluorescence changes. He gave a complete overview of the notable study published in 

 that further tested and validated the assay among a heterogenous group of synucleinopathies. Furthermore, he provided clinical context on the feasibility of the assay, the potential for clinical use, and the continued validation needed. 

Axsome to Submit NDA for AXS-05 in Alzheimer Agitation Following Positive Phase 3 Trials

FDA Clears IND for Trial Assessing Gene Therapy SGT-212 in Friedreich Ataxia

FDA Grants Fast Track Designation to Anti-Tau Therapy Posdinemab

 – Overviewing mechanistic function of SAAmplify-aSYN biomarker test, its purpose, and how it came about

 – Results from the published study; ways the fluorescence-based amplification method could distinguish MSA from PD and Lewy body dementia

 – Therapeutic feasibility of the assay in clinical settings and the next steps in validation

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Russell Lebovitz, MD, PhD. [LISTEN TIME: 22 minutes]

Episode 132: Aiding Diagnosis of Synucleinopathies Through SAAmplify-aSYN Biomarker Test

Episode 131, "Neurology Unwrapped: 2024's Most Intriguing Conversations" compiles some of the most compelling conversations from 

 podcast episodes throughout 2024. These discussions spotlight groundbreaking research and advancements in neurology that are propelling the field forward. The 

 team also explored the impact of several newly FDA-approved therapies, offering clinicians valuable insights into what these treatments mean for patient care and their practical use in the clinic. Featured in this week’s episode, in order of appearance, are:


, staff member of the Cleveland Clinic Mellen Center for Multiple Sclerosis, gives an overview on the new 2024 McDonald criteria to diagnose multiple sclerosis (MS), which was presented at the 

40th Congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS)

Understanding Major Changes to New McDonald Criteria for Multiple Sclerosis

, executive director of the leaders Engaged on Alzheimer's Disease (LEAD) coalition, on the changing and exciting advances in Alzheimer disease therapeutics, as well as the discontinuation of aducanumab, the first approved antiamyloid therapy.

Lessons Learned in Alzheimer Drug Development

, vice chair of the American Academy of Sleep Medicine's Practice Guidelines Task Force, discusses the newly revised guidelines for restless legs syndrome, including the latest updates in literature and changes to clinical care.

Implications of the 2024 AASM Guidelines for Restless Legs Syndrome

, a movement disorder specialist at the Marcus Neuroscience Institute of Baptist Health, provided clinical perspective on the use of the Syn-One diagnostic test for patients with Parkinson disease.

Utilizing the Syn-One Test to Diagnose Parkinson Disease

, an assistant professor of neurosurgery at Mayo Clinic Arizona, spoke at the 2024 American Epilepsy Society Annual Meeting on cell therapy approaches for epilepsy, the challenges the clinical community faces, and the outlook for the future.

The Promise Behind Cell Therapy Approaches in Epilepsy

, a senior scientist at Sunnybrook Research Institute, gives insight on educating the next generation of neuromuscular and electrodiagnostic (EDX) practitioners, and the major differences in how this generation of medical students learn relative to previous ones.

Adapting Neuromuscular and Electrodiagnostic Medicine Education for Modern Learners

 – Ontaneda on the incorporation of new biomarkers into the 2024 McDonald Criteria for multiple sclerosis.

 – Kremer on the lessons learned from drug development, including antiamyloid treatments, and initial reaction to the discontinuation of aducanumab for AD. 

 – Berkowski on the outlook of treating and preventing restless legs syndrome going forward using the newly updated American Academy of Sleep Medicine treatment guidelines. 

 – Husain-Wilson on the function of the Syn-One test for Parkinson disease, its applicability, and how it may distinguish patients with PD from other synucleinopathies.

 – Parker on the promise and thought process behind stem cell approaches for drug-resistant epilepsy, including the different types of cell methods being tested. 

 – Robinson on the distinct differences in learning styles based on generation, the importance of adaptive teaching, and the new approaches educators in neuromuscular and electrodiagnostic medicine can take. 

Mind Moments®, a podcast from NeurologyLive®, brings you a review of 2024, with insights from Daniel Ontaneda, MD, PhD; Ian Kremer; Andy Berkowski, MD, PhD; Sameea Husain-Wilson, DO; Jonathan Parker, MD, PhD; and Lawrence Robinson, MD. [LISTEN TIME: 25 minutes]

Neurology Unwrapped: 2024’s Most Intriguing Conversations

In this episode, "Implications of the 2024 AASM Guidelines for Restless Legs Syndrome" Andy Berkowski, MD, PhD, founder of Relax Health, sat down to discuss the American Academy of Sleep Medicine's (AASM) recently published guideline update for the treatment of restless legs syndrome (RLS). Berkowski, who serves as a vice chair of the AASM's Practice Guidelines Task Force, provided clinical insight on the reasons behind the guidelines, pointing to the vast research advances and literature updates since its last iteration in 2012. Additionally, he touched on the greatest changes to the guidelines, noting things like the shift away from dopaminergic agents, the importance of iron in RLS pathophysiology, and the use of alpha-2-delta ligands and intravenous iron as first-line treatments. Berkowski also shared thoughts on how the guidelines impact care for patients of all ages, sexes, and RLS subtypes, as well as some of the more troubling parts of the guidelines to write and areas that were left unanswered. Furthermore, he gave his thoughts on how these guidelines will transform care going forward, including accelerating conversations on preventing RLS in many cases in the near future.

Neurogene Reports Serious Adverse Event in Phase 1/2 Rett Study of Gene Therapy NGN-401

Huntington Agent SAGE-718 to be Discontinued Following Disappointing Phase 2 DIMENSION Trial Results

Alzheimer Agent Simufilam Fails to Meet Primary End Point in Phase 3 Study

 – Reasons behind new guidelines, progress in clinical research

 – Overview of the greatest changes to the guidelines and what treating physicians should key in on

 – How the guidelines address management based on age, sex, and RLS subtype

 – Challenges when drafting the guidelines and the unanswered questions that remain

 – A promising future for treating, managing, and preventing RLS

REFERENCE
1. Winkelman JW, Berkowski JA, DelRosso LM, et al. Treatment of restless legs syndrome and periodic limb movement disorder: an American Academy of Sleep Medicine clinical practice guideline. J Clin Sleep Med. Published online September 26, 2024. doi:10.5664/jcsm.11390

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Andy Berkowski, MD, PhD. [LISTEN TIME: 26 minutes]

Episode 129: Implications of the 2024 AASM Guidelines for Restless Legs Syndrome

In this episode, "Adapting Neuromuscular and Electrodiagnostic Medicine Education for Modern Learners" Lawrence Robinson, MD, a senior scientist at Sunnybrook Research Institute, sat down to discuss his presentation from the 

2024 American Association of Neuromuscular and Electrodiagnostic Medicine (AANEM) annual meeting

, focused on educating the next generation of neuromuscular and electrodiagnostic (EDX) practitioners. Robinson gave an overview of his presentation, why this was a topic of interest, and the major differences in how this generation of medical students learn relative to previous ones. In addition, he discussed adapting to new learning styles, the benefits of flipped classrooms, and the impact of technology on education and practice. Furthermore, he touched upon the personal connections and humor in teaching, as well as ways to improve critical evaluation skills as a learner.

Phase 3 ENSURE Program of Vidofludimus Calcium Continues Following Positive Futility Analysis

Gene Therapy FLT201 Shows Promise in Early-Stage Study of Gaucher Disease

Risk of ARIA-E in Donanemab Attenuated Through New Enhanced Titration Method of Delivery

 – Overviewing and background on AANEM presentation

 – Challenges with educating next generation of neuromuscular and EDX practitioners

 – Areas of opportunity and growth for these next generation learners

 – Novelty and advantages of flipped classroom approaches to teaching

 – Future expected changes to neuromuscular care

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Lawrence Robinson, MD. [LISTEN TIME: 15 minutes]

Neuromuscular

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