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Articles

Marketed as Amvuttra, the Alnylam Pharmaceuticals RNA-interference treatment is now approved for an additional patient population of 150,000 in the US, and more than 300,000 worldwide.

FDA Approves Vutrisiran for ATTR-CM, Expanding Indication in Amyloidosis

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.

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

Videos

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]

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

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.

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

Marco Meglio, Assistant Managing Editor for 

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The MyacarinG study post-hoc analysis demonstrated rozanolixizumab's potential to significantly reduce ocular symptoms in gMG, with consistent improvements across multiple scoring systems.

Ocular Symptoms of Myasthenia Gravis Improved With Rozanolixizumab

Kathleen Costello discusses her new role as interim CEO of the CMSC. She has the support of Jeff Wilken, CMSC president, and Tina Trott, CMSC director.

Continuing the Vision of June Halper: What's Next for CMSC

Interim chief executive officer of the Consortium of Multiple Sclerosis Centers.

The interim chief executive officer at the Consortium of Multiple Sclerosis Centers invites healthcare professionals to the Consortium’s annual meeting from May 28–31 in Phoenix, Arizona. [WATCH TIME: 2 minutes]

What to Expect From the 2025 CMSC Annual Meeting: Kathleen Costello, CRNP, MSCN

The child neurologist and neuroimmunologist at Nationwide Children’s Hospital delved into the unique challenges and research objectives surrounding MOG antibody-associated disease. [WATCH TIME: 3 minutes]

Challenges and Research Priorities for Pediatric MOGAD: Kelsey Poisson, MD

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

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Nearly a decade in the making, revised guidelines provide 8 key points for effective nonpharmacologic treatment of epilepsy in childhood.

Clinical

Updated Clinical Guidelines on the Ketogenic Diet in Children with Epilepsy