Since the publication of the Duchenne muscular dystrophy (DMD) care considerations in
2010, multidisciplinary care of this severe, progressive neuromuscular disease has evolved …

Most rare diseases still lack approved treatments despite major advances in research
providing the tools to understand their molecular basis, as well as legislation providing …

Abstract Viltolarsen (Viltepso® in Japan) is a phosphorodiamidate morpholino antisense
oligonucleotide being developed by Nippon Shinyaku, in collaboration with the National …

Muscular dystrophies are a heterogeneous group of genetic diseases, characterized by
progressive degeneration of skeletal and cardiac muscle. Despite the intense investigation …

Duchenne muscular dystrophy (DMD) is a degenerative lethal, X-linked disease of skeletal
and cardiac muscles caused by mutations in the dystrophin gene. Cell therapy using …

Background Muscle wasting and weakness in Duchenne muscular dystrophy (DMD) causes
severe locomotor limitations and early death due in part to respiratory muscle failure. Given …

Background Duchenne muscular dystrophy (DMD) is a severe X-linked neuromuscular
childhood disorder that causes progressive muscle weakness and degeneration and results …

Muscular dystrophies are debilitating disorders that result in progressive weakness and
degeneration of skeletal muscle. Although the genetic mutations and clinical abnormalities …

Duchenne muscular dystrophy (DMD) is the most common childhood muscular dystrophy
affecting~ 1: 5000 live male births. Following the identification of pathogenic variations in the …

Duchenne muscular dystrophy (DMD) is a fatal genetic disorder characterized by
progressive muscle wasting that has currently no cure. Exon-skipping strategy represents …