Latest Conference Coverage

Remote Screening for Early Alzheimer’s Holds Promise Amid Key Challenges

Recently presented at the 2025 AD/PD Conference, the READL AD study aims to assess the feasibility of blood biomarkers and digital cognitive testing for early detection of Alzheimer disease.

Alzheimer Biomarkers pTau217 and pTau181 Show High Group-Level Correlation With Trial End Points

Investigators at AD/PD 2025 presented new data showing that changes in plasma p-tau217 and p-tau181 closely correlate with clinical outcomes in anti-amyloid therapy trials for Alzheimer disease.

Overviewing Design and Topline Data From Phase 3 APOLLOE4 Study of Valiltramiprosate in Alzheimer Disease: Anton Porsteinsson, MD

The director of the Alzheimer’s Disease Care, Research, and Education Program at the University of Rochester provided clinical insight on a pivotal trial testing a novel therapeutic in patients with Alzheimer disease who are carriers of APOE4/4. [WATCH TIME: 7 minutes]

NeuroVoices: Cynthia Lemere, PhD, on Exploring the Role of Complement in ARIA and CAA

The professor of neurology at Brigham and Women’s Hospital gave clinical insights on the therapeutic potential of targeting compliment as a way to mitigate risk for amyloid-related imaging abnormalities from antiamyloid therapies.

Genetic Risk Factors Influence Age at Onset in Autosomal Dominant Alzheimer Disease

A recent study presented at the 2025 AD/PD Conference identified key modifiers of Alzheimer disease onset in amyloid precursor protein duplication carriers.

Xanamem Shows Potential in Slowing Alzheimer Progression in Patients With Elevated Plasma pTau181

A phase 2a biomarker trial of xanamem presented at AD/PD 2025 suggests the drug may help slow clinical decline in patients with Alzheimer who have elevated levels of plasma pTau181.

Therapeutic Potential of Silmitasertib Observed in Huntington Disease Mouse Model

A recent study reported that silmitasertib, an investigational drug currently in development for cancer, may help reduce motor deficits and neuropathology in a Huntington disease.

Valiltramiprosate Demonstrates Greater Promise in Earlier Stages of Alzheimer Disease, Phase 3 APOLLOE4 Study Shows

Although valiltramiprosate failed to distinguish itself from placebo on the primary end point, the drug performed significantly better among mild MCI participants vs those with mild Alzheimer disease.

Higher CSF Aß42/Total Tau Ratio Associated With Increased ARIA Risk in Lecanemab

Research suggests that CSF ATI ratios could serve as a biomarker for identifying patients with Alzheimer disease at higher risk of ARIA during lecanemab treatment, aiding in safer patient management.

Exploring Multimodal Approaches to Neural Regeneration: Kuldip Dave, PhD

The senior vice president of research at The ALS Association talked about a multidisciplinary session presented at the 2025 MDA Conference that highlighted diverse strategies for neural repair, from pharmacological targets to noninvasive brain stimulation. [WATCH TIME: 6 minutes]

Insights into OPMD and Advancements in Genetic Research: Matthew Wicklund, MD

The professor of neurology at the University of Texas Health Science Center San Antonio provided clinical insights on the genetic basis of oculopharyngeal muscular dystrophy, as well as the primary challenges in diagnosing and treating the condition. [WATCH TIME: 5 minutes]

The Urgency of Funding and Equity in Neuromuscular Research and Care: Robert Califf, MD

The former FDA commissioner spoke on the need for policy and funding efforts for neuromuscular disorders, the stress on the clinical care system, and the need to acknowledge healthcare inequities. [WATCH TIME: 4 minutes]

NeuroVoices: Kuldip Dave, PhD, on Exploring Neural Regeneration in ALS and Beyond

The senior vice president of research at The ALS Association discussed insights into neural regeneration therapies and their potential to restore function in neurodegenerative diseases.

Exploring New Avenues for Treating Muscular Dystrophies With Targeted Therapies: Matthew Alexander, PhD

The associate professor of pediatric neurology and genetics at the University of Alabama Birmingham provided clinical insights on the key signaling pathways currently being targeted in the treatment of muscular dystrophies. [WATCH TIME: 4 minutes]

Differentiating Cognitive Decline in Multiple Sclerosis and Alzheimer Disease: Sarah Levy, PhD

The assistant professor in the department of neurology at Mount Sinai discussed distinguishing cognitive impairment in MS from AD emphasizing orientation as a key differentiator. [WATCH TIME: 5 minutes]

Duchenne Gene Therapy Delandistrogene Moxeparvovec Shows Manageable Safety Over 5-Year Period

Pooled safety data of delandistrogene moxeparvovec, a gene transfer therapy for Duchenne muscular dystrophy, suggests a manageable tolerability profile up to 5 years of follow-up.

Challenges and Trust in Telehealth Integration for Multiple Sclerosis Care: Enrique Alvarez, MD, PhD

The professor of neurology at University of Colorado School of Medicine talked about the challenges of telehealth and device-based data collection in clinical practice. [WATCH TIME: 4 minutes]

Friedreich Ataxia Therapy Nomlabofusp Increases Frataxin Levels in Phase 2 Study

Findings from a phase 2 study of nomlabofusp, a novel investigational therapy for Friedreich ataxia, demonstrated an increase in tissue frataxin levels and metabolic improvements.

Addressing Persistent Challenges in NMOSD Diagnosis and Treatment: Sumaira Ahmed

Despite advancements in NMOSD treatments, the founder and executive director of the Sumaira Foundation discussed how misdiagnosis, delayed diagnosis, and disparities in care remain significant challenges. [WATCH TIME: 4 minutes]

Short-Term Analysis Confirms Safety of Oral Edaravone in ALS Over 96 Weeks

Results from a recent phase 3 study presented at the 2025 MDA Conference reported that oral edaravone remained well tolerated in patients with ALS over 96 weeks, with no new safety concerns.

MDA Study Highlights Lack of Psychosocial Care Services for Duchenne Muscular Dystrophy

Despite facing mental health concerns with anger, aggression, or irritability, among others, slightly less than one-fourth of patients with DMD utilized psychosocial services such as counseling or therapy.

Sevasemten Reduces Muscle Injury Biomarkers of Becker Muscular Dystrophy in Latest Analysis

The phase 2 CANYON trial findings presented at the 2025 MDA conference highlight sevasemten’s potential in reducing muscle injury biomarkers in Becker muscular dystrophy.

Updates on Guidance for Diagnosing Pediatric and Late-Onset Multiple Sclerosis: Andrew Solomon, MD

The professor of neurological sciences at the University of Vermont discussed the work of the MS Differential Diagnosis Consortium, highlighting efforts to refine MS differential diagnosis and improve clinical accuracy. [WATCH TIME: 8 minutes]

Celebrating Milestones and Tackling New Frontiers in Pompe Disease: Priya Kishnani, MD

The professor of pediatrics and division chief of Medical Genetics at Duke University gave clinical insight on a presentation on the advancements and limitations of enzyme replacement therapy for Pompe Disease. [WATCH TIME: 3 minutes]

Episode 137: Advancing Neuromuscular Care and Research

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

Gene Therapy RGX-202 Shows Positive Interim Results in Duchenne Muscular Dystrophy Trial

New biomarker data presented at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference of RGX-202 in the phase 1/2 trial highlight strong microdystrophin expression.

Using Baseline Paramagnetic Rim Lesion Count as Prognostic and Treatment Biomarkers in Multiple Sclerosis: Jiwon Oh, MD, PhD

The medical director of the Barlo Multiple Sclerosis Program at St Michaels Hospital discussed findings from phase 3 trials suggesting that baseline PRLs can predict disability accumulation and may serve as biomarkers for treatment response to tolebrutinib. [WATCH TIME: 6 minutes]

Givinostat Delays Duchenne Muscular Dystrophy Progression by 2 Years, Simulation Model Shows

A new analysis reported that givinostat, an oral histone deacetylase inhibitor recently approved for DMD, slowed disease progression by approximately 2 years compared with standard care.

Critical Lessons From Emergence of Enzyme Replacement Therapies in Pompe Disease: Priya Kishnani, MD

The professor of pediatrics and division chief of Medical Genetics at Duke University delved into the transformative impact of enzyme replacement therapy on Pompe disease, addressing its advancements, limitations, and promising innovations shaping its future. [WATCH TIME: 2 minutes]

Breaking Barriers: Donovan Decker’s Journey in Gene Therapy and Advocacy for LGMD

Donovan Decker, recipient of the 2025 MDA Legacy Award for Community Impact and Research, shared his powerful journey as a patient advocate and gene therapy pioneer, shedding light on the challenges and progress in LGMD.