… effective treatments for patients with Duchenne muscular dystrophy. Many recent studies have
… New therapies for Duchenne muscular dystrophy: challenges, prospects and clinical trials. …

… In neuromuscular disorders such as Duchenne muscular dystrophy (DMD), the pathological
consequences of the lack of dystrophin and the loss of the dystrophin-associated protein …

… Abstract: In recent years, antisense oligonucleotide (ASO) has been very active in … in
Duchenne muscular dystrophy, where it made a major breakthrough. Duchenne muscular dystrophy …

… breathing in children suffering form Duchenne muscular dystrophy (DMD). Because of the
unique physiological alterations during sleep, respiratory muscle dysfunction in DMD may be …

… and inotersen) have been clinically used for the treatment of spinal muscular atrophy,
Duchenne muscular dystrophy, and hereditary transthyretin amyloidosis. Apart from these, …

… Duchenne muscular dystrophy improved by … Medicines Agency review of ataluren for the
treatment of ambulant patients aged 5 years and older with Duchenne muscular dystrophy …

… ) and muscle cell membrane through the protein complex. Lack of dystrophin will lead to
Duchenne muscular dystrophy (DMD), which features muscle weakness and muscle …

… I will refer to the Duchenne example to highlight these features. Duchenne muscular dystrophy
(DMD) is a progressive and fatal rare X-linked neuromuscular monogenetic disease with …

… Ultrasound assessment of diaphragm function in pediatric patients with Duchenne muscular
dystrophy[J]. Chinese Journal of Medical Ultrasound (Electronic Edition), 2023, 20(10): 1068…

… Duchenne型肌营养不良（duchenne muscular dystrophy, DMD）是进行性肌营养不良中最常见
的类型，以进行性、致死性为主要特点。世界上每3 500~6 000个新生男婴中就有一名DMD患儿 […