Rare Disease Report

Imaging Biomarkers in Duchenne Muscular Dystrophy

APRIL 28, 2015


Kenneth Fischbeck, MD, of the National Institute of Neurological Disorders and Stroke (NINDS) talks about the recent study he and his colleagues performed to determine if non invasive measures could be used to measure treatment efficacy in boys with Duchenne muscular dystrophy receiving exon-skipping therapy.

Reference

Mankodi A,Janiczek R,Noura Azzabou N, et al. Skeletal muscle, cardiac, and pulmonary imaging biomarkers of disease activity in a trial of exon-skipping for boys with Duchenne muscular dystrophy. Presented at: American Academy of Neurology Annual Meeting; April 18-25, 2015; Washington, DC. Abstract P5.052.

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