The increased understanding of the genes and pathogenic mechanism of the LGMDs will
improve diagnostic processes and prognostic accuracy, and promote therapeutic strategies …

Background We report clinical safety and biochemical efficacy from a dose-ranging study of
intravenously administered AVI-4658 phosphorodiamidate morpholino oligomer (PMO) in …

Background Mutations that disrupt the open reading frame and prevent full translation of
DMD, the gene that encodes dystrophin, underlie the fatal X-linked disease Duchenne …

Background Duchenne muscular dystrophy (DMD) is a severe, progressive, and rare
neuromuscular, X-linked recessive disease. Dystrophin deficiency is the underlying cause of …

Children with Duchenne muscular dystrophy (DMD) are shorter than their healthy peers. The
introduction of corticosteroid (CS) has beneficial effects on muscle function but slows growth …

Background Duchenne muscular dystrophy is caused by dystrophin deficiency and muscle
deterioration and preferentially affects boys. Antisense-oligonucleotide-induced exon …

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

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 …

Limb girdle muscular dystrophies (LGMD) are characterized by genetic and clinical
heterogeneity: seven autosomal dominant and 12 autosomal recessive loci have so far been …

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 …