Neuromuscular

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Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending August 1, 2025.

Capricor Therapeutics navigates FDA challenges for deramiocel, aiming to advance treatment for DMD-related cardiomyopathy with promising trial data.

Roche faces regulatory challenges for Elevidys, an approved gene therapy for Duchenne muscular dystrophy, amid safety concerns and clinical trial setbacks.

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A new phase 3 study will explore masitinib's potential as treatment for ALS, aiming to enhance patient outcomes and confirm earlier promising results.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending July 25, 2025.

AstraZeneca's gefurulimab shows promising results in treating generalized myasthenia gravis, offering patients a convenient, self-administered therapy option.

Neurology experts anticipate groundbreaking clinical trial results in 2025, potentially transforming treatment strategies for various neurological conditions.

Members from Nuvig Therapeutics discussed the rationale and therapeutic development of NVG-2089, a novel anti-inflammatory agent being studied for chronic inflammatory demyelinating polyneuropathy.

The FDA-cleared duo devices allow for a more simplified administration of the liquid medicine Hyqvia, reducing the number of steps needed to prepare for the infusion of 2 dual vial units or more.

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FDA halts Sarepta's Elevidys gene therapy distribution after three patient deaths, prioritizing safety amid ongoing clinical trial evaluations.

Elisabeth Marsh, MD, an associate professor of neurology at Johns Hopkins, shared her insights on the emerging role of AI in clinical practice and neurologic education, ahead of her session at the 2025 ANA/AUPN Annual Meetings.

Nuri Jacoby, MD, neurologist at Maimonides Medical Center and SUNY Downstate, previewed a hands-on session at the upcoming AUP Annual Meeting exploring how simulations can teach communication, professionalism, and feedback skills in neurology training.

Nester Tomycz, MD, director of neurosurgical pain division at Allegheny General Hospital, discussed emerging neurosurgerical approaches for chronic pain, with a focus on neuromodulation, patient selection, and non-opioid treatment pathways.

Recently reported phase 2b results revealed that PrimeC consistently altered ALS-related miRNA expression, reinforcing its potential as a multi-targeted therapy and aligning with previously reported clinical benefits.

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Indirect treatment comparisons showed significantly greater or statistically higher improvements in MG-ADL scores with nipocalimab versus other FcRn blockers over 24 weeks.

Denali Therapeutics advances treatment for Hunter syndrome with FDA's priority review of tividenofusp alfa, targeting brain delivery and improved patient outcomes.

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Catch up on any of the neurology news headlines you may have missed over the course of June 2025, compiled all into one place by the NeurologyLive® team.

New findings reveal deramiocel's promising long-term benefits for boys with Duchenne muscular dystrophy cardiomyopathy, enhancing cardiac function and muscle performance.

New phase 1 data revealed salanersen's promise to slow neurodegeneration in spinal muscular atrophy, potentially paving the way for future phase 3 trials.

Edgewise Therapeutics reveals promising results for sevasemten, potentially the first approved treatment for Becker muscular dystrophy, enhancing patient outcomes.

Overviewing the major clinical trial readouts in neurology from the first half of 2025, with data that could potentially reshape patient care.

Here's some of what is coming soon to NeurologyLive® this week.

Efgartigimod alfa SC is now approved in Europe for patients with chronic inflammatory demyelinating polyneuropathy, following a positive recommendation from the Committee for Medicinal Products for Human Use.

Here's some of what is coming soon to NeurologyLive® this week.

Test your neurology knowledge with NeurologyLive®'s weekly quiz series, featuring questions on a variety of clinical and historical neurology topics. This week's topic is on pediatric neurology.