Treatment Guidelines and Rehabilitation in Spinal Muscular Atrophy and Duchenne's Muscular Dystrophy

Authors

Eleni Drakou, Saul R Korey Department of Neurology, Montefiore Medical Center, Albert Einstein College of Medicine, 111 East 210th Street, Bronx, NY 10467, USA. Electronic address: elendrakou@gmail.com.
Sarah Wright, Department of Neurology and Rehabilitation, George Washington University School of Medicine, Children's National Hospital, 111 Michigan Avenue Northwest, Washington, DC 20010, USA; Department of Pediatrics, George Washington University School of Medicine, Children's National Hospital, 111 Michigan Avenue Northwest, Washington, DC 20010, USA.
Leslie D. Delfiner, Isabelle Rapin Division of Child Neurology, Saul R Korey Department of Neurology, Albert Einstein College of Medicine, MDA Care Center at Montefiore, 3415 Bainbridge Avenue, Bronx, NY 10467, USA.
David Cancel, Department of Rehabilitation and Human Performance, Pediatric Rehabilitation Medicine, Icahn School of Medicine at Mount Sinai, 3 East 101st Street, New York, NY 10029, USA; Department of Pediatrics, Pediatric Rehabilitation Medicine, Icahn School of Medicine at Mount Sinai, 3 East 101st Street, New York, NY 10029, USA.

Document Type

Journal Article

Publication Date

8-1-2025

Journal

Physical medicine and rehabilitation clinics of North America

Volume

36

Issue

3

DOI

10.1016/j.pmr.2025.03.001

Keywords

DMD; Duchenne muscular dystrophy; Muscular dystrophy; Neuromuscular disorders; SMA; Spinal muscular atrophy

Abstract

Duchenne muscular dystrophy (DMD) and spinal muscular atrophy (SMA) are childhood-onset neuromuscular disorders with substantial morbidity and significant rehabilitation needs. Recent advancements, particularly gene-based therapies, have significantly altered the disease trajectory for both conditions, necessitating updated care guidelines. This review will delve into the pathogenesis of DMD and SMA, explore the latest disease-modifying treatments available for each, and comprehensively address the multifaceted rehabilitation needs across the spectrum of disease severity. The aim is to provide a comprehensive overview of current best practices to optimize functional outcomes and quality of life for individuals with DMD and SMA.

APA Citation

Drakou, Eleni; Wright, Sarah; Delfiner, Leslie D.; and Cancel, David, "Treatment Guidelines and Rehabilitation in Spinal Muscular Atrophy and Duchenne's Muscular Dystrophy" (2025). GW Authored Works. Paper 7858.
https://hsrc.himmelfarb.gwu.edu/gwhpubs/7858

Department

Neurology