As one of the most severe forms of muscle dystrophy, Duchenne muscular dystrophy (DMD)
results in progressive muscle wasting, ultimately resulting in premature death due to …

Duchenne muscular dystrophy (DMD) is an X-linked genetic disease primarily affecting boys
causing loss of the dystrophin protein, ultimately leading to muscle wastage and death by …

Duchenne muscular dystrophy (DMD) is a severe, progressive neuromuscular disorder
caused by reading frame disrupting mutations in the DMD gene leading to absence of …

Duchenne muscular dystrophy (DMD) is caused by X-linked inherited or de novo DMD gene
mutations predominantly affecting males who develop early-onset muscle degeneration …

Background Oligonucleotide therapeutics have emerged as a promising and dynamic class
of pharmaceutical agents with remarkable potential for treating a wide spectrum of genetic …

ABSTRACT Introduction: Recessive Dystrophic Epidermolysis Bullosa (RDEB) is a
monogenetic inherited genodermatosis associated with deleterious mutations in the gene …

Neurofibromatosis type 1 (NF1) is an autosomal dominant genetic disorder with almost 3000
different disease-causing variants within the NF1 gene identified. Up to 44% of these …

Conventionally, the gold standard of DMD drug therapy is the administration of
glucocorticoid steroids, either prednisone or deflazacort, that are routinely prescribed to …

Abstract Duchenne Muscular Dystrophy (DMD) is one of the most common inherited
disorders worldwide. As there is currently no absolute treatment, the present systems …

Duchenne muscular dystrophy (DMD) is a severe, progressive neuromuscular disorder
caused by reading frame disrupting mutations in the DMD gene leading to absence of …