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 …

Decades of research in skeletal muscle physiology have provided multiscale insights into
the structural and functional complexity of this important anatomical tissue, designed to …

Abstract Background Duchenne Muscular Dystrophy (DMD) is a rare disorder caused by
mutations in the dystrophin gene. A recent systematic review and meta-analysis of global …

Antisense oligonucleotides (ASOs) were first discovered to influence RNA processing and
modulate protein expression over two decades ago; however, progress translating these …

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 …

Background A variety of mutations in the largest human gene, dystrophin, cause a spectrum
from mild to severe dystrophin-associated muscular dystrophies. Duchenne (DMD) and …

Objective To report safety, pharmacokinetics, exon 53 skip**, and dystrophin expression
in golodirsen-treated patients with Duchenne muscular dystrophy (DMD) amenable to exon …

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 …

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 …