NeurologyLive

11/15/2025

🎙️ A new Mind Moments® episode just dropped!
Episode 154: NEALS 2025: Takeaways That Matter for ALS Care

Experts Dr. Jinsy Andrews and Dr. James Berry break down the biggest updates from the 2025 NEALS Meeting, including new gene therapy approaches, progress from the HEALEY Platform Trial, MY-MATCH, SOD1 research, and major changes improving access to ALS clinical trials.

They also look ahead at what’s coming in 2026—from digital endpoints to new collaborations shaping the future of ALS care.

If you follow ALS, neurology news, or medical innovation, this is a great episode to dive into.

🎧 Listen now: https://hubs.li/Q03TcPG_0

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with James Berry, MD, MPH; and Jinsy Andrews, MD, MSc. [LISTEN TIME: 28 minutes]

11/14/2025

🚨Today, the U.S. Food and Drug Administration approved safety-related labeling updates for delandistrogene moxeparvovec-rokl (Elevidys), a marketed gene therapy for , to include a new boxed warning and a restriction limiting its treatment use for ambulatory patients ages 4 and older with the condition. Sarepta Therapeutics

View the update here:

The agency has required a postmarketing observational study to monitor safety for at least 12 months following gene therapy treatment in patients with Duchenne muscular dystrophy.

11/14/2025

James F. Howard Jr, MD, FAAN; Beth Stein, MD; Andrew Gordon, MD; and Ratna Kiran Bhavaraju-Sanka, MD, explore how the treatment of myasthenia gravis (MG) is evolving from conventional symptomatic therapies toward more targeted and personalized approaches. The discussion highlights how early intervention, shared decision-making, and a focus on achieving minimal symptom expression are redefining standards of care for patients.

In recent Peer Exchange episodes, these experts address the current standard of care and the ongoing challenges of treatment burden, emphasizing the need to better align therapeutic goals with patient quality of life. They examine how targeted therapies—such as FcRn inhibitors, complement inhibitors, and B cell–directed options like inebilizumab—are transforming disease management and addressing key unmet needs in MG care.

The conversation also explores the clinical rationale for sequencing targeted therapies and the growing role of mechanism-based strategies in improving long-term outcomes. From early intervention to emerging therapies, the panel provides valuable clinical pearls and practical insights to guide more effective, individualized management of myasthenia gravis.

Watch Now: https://hubs.li/Q03T60R80

11/14/2025

In a recent interview, Paul Melmeyer, MPP, executive vice president of public policy and advocacy at the Muscular Dystrophy Association, discussed how collaboration can improve policy, funding, and care for patients with diseases.

Watch Now: https://hubs.li/Q03T75sN0

11/14/2025

Big progress for the LGMD2I/R9 community 💪

BridgeBio announced positive phase 3 results for BBP-418, a potential first-in-class oral therapy. The FORTIFY study showed significant improvements in movement, breathing, and muscle function, along with strong biomarker results and no new safety concerns.

The company plans to submit a New Drug Application (NDA) to the FDA in 2026, aiming to make BBP-418 the first approved treatment for LGMD2I/R9.

This marks an important step forward for people living with limb-girdle muscular dystrophy type 2I/R9 and their families. 💙

Check it out: https://hubs.li/Q03T5yBX0

The phase 3 FORTIFY trial of BBP-418 met its primary and key secondary interim analysis end points and demonstrated a safety profile consistent with prior studies in patients with LGMD2I/R9.

11/13/2025

Hope for people living with Parkinson 💙

Early results from UX-DA001, a new stem cell–based therapy, show better motor control and dopamine activity—without serious side effects. This breakthrough brings us closer to repairing the brain using a patient’s own cells.

🧬 A promising sign for the future of personalized medicine and neuroregeneration.

👉 Read more about this exciting development: https://hubs.li/Q03T0j8Y0

Liche Zhou, MD, of the department of neurology at Jiang University School of Medicine, provided commentary on new phase 1 data on an iPSC-targeted cell therapy presented at the 2025 MDS Congress.

11/13/2025

Promising Results for a New Narcolepsy Treatment 🧠💤

The phase 2 VIBRANCE-2 trial found that alixorexton (ALKS 2680)—a once-daily oral orexin-2 receptor agonist—significantly improved wakefulness and daytime sleepiness in people with narcolepsy type 2.

✨ Strong efficacy at higher doses
✨ Well-tolerated with no serious side effects
✨ Nearly all participants joined the open-label extension

“These data represent an important breakthrough for patients,” said Dr. Emmanuel Mignot, Stanford University.

With phase 3 trials ahead, alixorexton could bring a much-needed new option for those living with narcolepsy.

Read more: https://hubs.li/Q03SVwnv0

New trial results reveal alixorexton's significant efficacy in treating narcolepsy type 2, marking a potential breakthrough in sleep medicine.

11/13/2025

💊 New hope for people living with multiple sclerosis!

Genentech’s investigational drug fenebrutinib has shown strong results in two major phase 3 trials, helping reduce relapses in relapsing MS and slow disability progression in primary progressive MS.

This could mark the arrival of a new class of oral therapies that work by blocking BTK — a protein that helps drive inflammation in MS.

As Dr. Levi Garraway from Genentech said, this approach may become an “important new treatment option” for people with both forms of MS.

Read more here: https://hubs.li/Q03SSzMw0

Findings from a second phase 3 trial, dubbed FENhance 1, assessing fenebrutinib in patients with relapsing multiple sclerosis are anticipated by the first half of 2026.

11/13/2025

🌟 A historic step forward for families affected by TK2d!

For the first time ever, the FDA has approved a treatment for thymidine kinase 2 deficiency (TK2d) — a rare genetic condition that causes severe muscle weakness and respiratory problems.

The new therapy, Kygevvi, developed by UCB, has been shown to dramatically improve survival and bring renewed hope to families who previously had no treatment options.

As Fabian Somers, PhD of UCB shared:
“Kygevvi brings real hope — the gift of hope and the gift of time.” 💙

This approval is a victory for science, perseverance, and the global rare disease community.

Read about it ➡️ https://hubs.li/Q03SPnNr0

The vice president and Asset Head of Rare and Ultra-Rare Diseases at UCB provided clinical perspectives on the FDA approval of Kygevvi, the first marketed treatment specific for patients with thymidine kinase 2 deficiency (Tk2d).

11/13/2025

What can 30+ years of longitudinal data teach us about Parkinson disease and dementia?

G. Webster Ross, MD, breaks down the latest from the Honolulu-Asia Aging Study (HAAS)—a landmark project tracking disease risk in more than 3800 individuals since 1991.

🧠 Study implications
🩺 Autopsy-based insights
📊 Risk factor profiles

An investigator at Kuakini Health Systems spoke about the global importance of the Honolulu-Asia Aging Study, highlighting its history and findings, including data surrounding the diagnosis of neurodegenerative diseases. [WATCH TIME: 11 minutes]

11/12/2025

ICYMI: Axsome Therapeutics has submitted an sNDA for AXS-05, an oral NMDA antagonist/sigma-1 agonist for Alzheimer agitation.

Submission supported by data from ACCORD-2 and ADVANCE-2 trials.
🔖

Axsome Therapeutics advances AXS-05 for Alzheimer agitation, showcasing promising trial results and potential for treating major depressive disorder.

11/12/2025

It was a busy month of October for the FDA! Whether you're a clinician, industry leader, or researcher, take a peek at all the latest activity with our action update:

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Catch up on any of the neurology news headlines you may have missed in October 2025, compiled into 1 place by the NeurologyLive® team.