Duchenne muscular dystrophy is a severe, progressive, muscle-wasting disease that leads
to difficulties with movement and, eventually, to the need for assisted ventilation and …

Abstract Background Duchenne Muscular Dystrophy (DMD) is a rapidly progressive, lethal
neuromuscular disorder, present from birth, which occurs almost exclusively in males. We …

Analyzing the type and frequency of patient‐specific mutations that give rise to Duchenne
muscular dystrophy (DMD) is an invaluable tool for diagnostics, basic scientific research …

Current molecular genomic approaches to human genetic disorders have led to an
explosion in the identification of the genes and their encoded proteins responsible for these …

Provision of a molecularly confirmed diagnosis in a timely manner for children and adults
with rare genetic diseases shortens their" diagnostic odyssey," improves disease …

Background Duchenne muscular dystrophy (DMD) is a severe rare progressive inherited
neuromuscular disorder, leading to loss of ambulation (LOA) and premature mortality. The …

Objective: The objective of this study was to estimate the total cost of illness and economic
burden of Duchenne muscular dystrophy (DMD). Methods: Patients with DMD from …

Background: Recent short-term clinical trials in patients with Duchenne Muscular Dystrophy
(DMD) have indicated greater disease variability in terms of progression than expected. In …

Duchenne muscular dystrophy (DMD) is the most common form of muscular dystrophy in
childhood. It is caused by mutations of the DMD gene, leading to progressive muscle …

Duchenne muscular dystrophy (DMD) is a rare pediatric neuromuscular disease associated
with progressive muscle degeneration and extensive care needs. Our objective was to …