Duchenne Muscular Dystrophy

Variability in Cardiac Diagnostics and Therapies for Patients with Duchenne Muscular Dystrophy
Variability in Cardiac Diagnostics and Therapies for Patients with Duchenne Muscular Dystrophy 1024 670 Mary Bates, PhD

Survey shows different approaches to cardiac diagnostics and therapy among a multicenter collaborative network. Cardiac disease is a major cause of mortality in Duchenne muscular dystrophy (DMD), yet data regarding outcomes and cardiac disease progression are lacking. This has led to a highly variable approach to heart failure therapy in DMD from center to center,…

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Encouraging First Report of Systemic Delivery of Micro-dystrophin Gene Therapy in Children With Duchenne Muscular Dystrophy
Encouraging First Report of Systemic Delivery of Micro-dystrophin Gene Therapy in Children With Duchenne Muscular Dystrophy 150 150 Abbie Roth

One-year data from the first four patients to receive a single dose of the rAAVrh74.MHCK7.micro-dystrophin gene therapy is published in JAMA Neurology. Researchers from Nationwide Children’s Hospital have published in JAMA Neurology results from the first four patients treated in the first clinical trial of systemic delivery of micro-dystrophin gene therapy in children with Duchenne muscular dystrophy…

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