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Duchenne Muscular Dystrophy

FDA Approves Micro-dystrophin Gene Therapy for Duchenne Muscular Dystrophy
 FDA Approves Micro-dystrophin Gene Therapy for Duchenne Muscular Dystrophy 1024 632 Abbie Miller
By: Abbie Miller

On June 22, 2023, the FDA announced accelerated approval for SRP-9001/ELEVIDYS for Duchenne muscular dystrophy (DMD) following decades of research in the Center for Gene Therapy at Nationwide Children’s Hospital. The FDA approved the treatment for pediatric patients 4-5 years old with DMD. Jerry Mendell, MD, principal investigator in the Center for Gene Therapy at…

Micro-dystrophin: A Small Gene With Big Promise
 Micro-dystrophin: A Small Gene With Big Promise 1024 632 Abbie Miller
By: Abbie Miller

SRP-9001 for Duchenne muscular dystrophy supplies a functional dystrophin gene via AAVrh74 gene therapy. In 1969, Jerry Mendell, MD, was working at the National Institute of Neurological Disorders and Stroke (NINDS) when he saw his first patient with Duchenne muscular dystrophy (DMD). DMD, a severe form of muscular dystrophy caused by a mutation in the…

Proof-of-Concept Study Shows Promise of Exon-Skipping Gene Therapy Approach in Duchenne Muscular Dystrophy
 Proof-of-Concept Study Shows Promise of Exon-Skipping Gene Therapy Approach in Duchenne Muscular Dystrophy 1024 575 Lauren Dembeck
By: Lauren Dembeck

Duchenne muscular dystrophy (DMD) is a devastating progressive muscle-wasting disease that leads to loss of motor and cardiorespiratory function. The disease is caused by mutations in the DMD gene that result in the loss of expression of the dystrophin protein, which plays a critical role during contraction and relaxation of muscle. To date, the U.S.…

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
By: 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,…

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 Miller
By: Abbie Miller

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…