Tag: Duchenne


Encouraging First Report of Systemic Delivery of Micro-dystrophin Gene Therapy in Children with Duchenne Muscular Dystrophy

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...

UTSW/Children's Health joint program approved as Certified Duchenne Care Center

A joint program of UT Southwestern Medical Center and Children’s Health has been approved as a Certified Duchenne Care Center by Parent Project Muscular Dystrophy,...
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RNA-Seq Analysis of an Antisense Sequence Optimized for Exon Skipping in Duchenne Patients Reveals No Off-Target Effect

Domenger, C; Allais, M; François, V; Léger, A; Lecomte, E; Montus, M; Servais, L; … Le Guiner, C; + view all Domenger, C; Allais, M;...

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