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Chamindra Laverty, MD  (Credit: University of California, San Diego)
Givinostat Delays Duchenne Muscular Dystrophy Progression by 2 Years, Simulation Model Shows

March 20th 2025

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.

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

March 20th 2025

Pete Salzmann, MD, MBA  (Credit: LinkedIn)
Immunovant Reports Positive Phase 3 and Phase 2b Results for Batoclimab in Myasthenia Gravis and CIDP

March 19th 2025

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

March 19th 2025

Jennifer Kwon, MD, MPH  (Credit: University of Wisconsin)
Intrathecal Onasemnogene Abeparvovec Demonstrates Favorable Safety and Motor Function Stabilization in Treatment-Experienced Patients With SMA

March 19th 2025

Coverage of the 2025 ACTRIMS Forum

Get direct access to interviews with experts, as well as the latest data and clinical news updates, straight from the conference floor in West Palm Beach.

ACTRIMS 2025

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

March 20th 2025

Chamindra Laverty, MD  (Credit: University of California, San Diego)
Givinostat Delays Duchenne Muscular Dystrophy Progression by 2 Years, Simulation Model Shows

March 20th 2025

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

March 20th 2025

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

March 20th 2025

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