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 of micro-dystrophin gene therapy in children with Duchenne muscular dystrophy (DMD) – and initial findings suggest that the therapy can provide functional improvement that is greater than that observed under the standard of care.

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Duchenne Muscular Dystrophy Diagnosis Improved by Simple Accelerometers

Testing for Duchenne muscular dystrophy can require specialized equipment, invasive procedures and high expense, but measuring changes in muscle function and identifying compensatory walking gait could lead to earlier detection. This week in Chaos, researchers present a relative coupling coefficient, which can be used to quantify the factors involved in the human gait and more accurately screen for the disorder. They measured movements of different parts of the body in test subjects, viewing the body as a kinematic chain.

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