Jiya O. Chacko

A Registry-Based Perspective of Interventional Clinical Trials for Duchenne Muscular Dystrophy

Background and objective Duchenne muscular dystrophy (DMD) is a severe, progressive, X-linked genetic disorder caused by pathogenic variants in the dystrophin gene, leading to progressive muscle weakness, loss of ambulation, and premature mortality. Clinical trials are essential for advancing evidence-based management...