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 …

Duchenne muscular dystrophy (DMD) is a severe, progressive, and ultimately fatal disease
of skeletal muscle wasting, respiratory insufficiency, and cardiomyopathy. The identification …

Background and Objectives Duchenne muscular dystrophy (DMD) is a rare progressive
disease that is often diagnosed in early childhood and leads to considerably reduced life …

Rare diseases represent a highly heterogeneous group of disorders with high phenotypic
and genotypic diversity within individual conditions. Due to the small numbers of people …

Duchenne muscular dystrophy is an X-linked disease caused by the absence of functional
dystrophin in the muscle cells. Major advances have led to the development of gene …

Duchenne muscular dystrophy (DMD) is caused by the lack of functional dystrophin protein.
Improvements in patient care and disease management have slowed down disease …

Duchenne muscular dystrophy (DMD) is a rare, X-linked neuromuscular disease caused by
pathogenic variants in the DMD gene that result in the absence of functional dystrophin …

Background Duchenne muscular dystrophy, the most common childhood muscular
dystrophy, is caused by dystrophin deficiency. Preclinical and phase 2 study data have …

Importance Corticosteroidal anti-inflammatory drugs are widely prescribed but long-term use
shows adverse effects that detract from patient quality of life. Objective To determine if …

Duchenne muscular dystrophy (DMD) is a severe, progressive, and incurable X-linked
disorder caused by mutations in the dystrophin gene. Patients with DMD have an absence of …