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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]

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

Articles

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.

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

The company recently announced positive topline results from its phase 3 study in patients with MG and initial data from its phase 2b study in patients with CIDP, both assessing batoclimab. 

Immunovant Reports Positive Phase 3 and Phase 2b Results for Batoclimab in Myasthenia Gravis and CIDP

Two separate long-term extension studies met their pre-specified endpoints, showing highly significant evidence of sustained treatment benefits in slowing disease progression in pediatric and adult patients.

Investigational Vatiquinone Slows Friedreich Ataxia Disease Progression Relative to FACOMS Natural History Cohort

Marco Meglio, Assistant Managing Editor for 

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

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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We present a selection of interesting neurology articles for the month of January in the following slides. See the slide legends for links to the article summaries.

Slideshows

January news includes the first FDA-approved drug for spinal muscular atrophy, various epilepsy treatments & vitamin D’s impact on Alzheimer & headache.

Interesting Neuro: Epilepsy Outcomes & Vitamin D