Pediatric neurology
Duchenne Muscular Dystrophy: Clinical Innovations, Ethical Considerations, and Evolving the Path to Adulthood.
Natalie Kukulka, Paul Vermilion, Bo Lee
Published: 202510.1016/j.pediatrneurol.2025.11.020
Abstract
Duchenne Muscular Dystrophy (DMD) is a progressive and life-limiting neuromuscular disorder with profound implications for affected individuals and their families. Advances in genetic and molecular therapies, including exon skipping agents, gene ther…
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