Tag: Muscular

Pediatrics

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

Newsemia
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...
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German researchers recommend early screening and treatment for spinal muscular atrophy

Newsemia
The advent of therapeutic interventions for spinal muscular atrophy (SMA) has increased the importance of presymptomatic diagnosis and treatment. When to start treatment in children...

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