… of conditions that impair the function of muscles, motor neurons, peripheral … muscular
dystrophy, Duchenne muscular dystrophy (DMD), is caused by mutations in the X-linked dystrophin …

… 1A Phase 2 clinical trial evaluating the safety and efficacy of SRP-9001 for treating patients
with Duchenne muscular dystrophy. World Muscle Society Congress; Sept 20–24, 2021 (…

… Duchenne muscular dystrophy (DMD) is one of the most severe and devastating neuromuscular
hereditary diseases with a male newborn incidence of 20 000 cases each year. The …

… This highlights the need to fill the four-year gap to provide updated … The lack of an updated
systematic review and meta-analysis … The aim of this study is therefore to update the previous …

… The therapeutic landscape of muscular dystrophies has changed with the … treatments for
Duchenne muscular dystrophy (DMD), the most common and severe muscular dystrophy. This …

… improvements in the diagnosis and care of Duchenne muscular dystrophy (DMD), the most
common childhood muscular dystrophy. International guidelines have been published and …

… Ataluren is indicated for the treatment of Duchenne muscular dystrophy resulting from a
nonsense mutation in the dystrophin gene, in ambulatory patients aged 2 years and older in the …

… In June 2022, recent updates to this study expressed concern over vesleteplirsen after a
serious adverse event of hypomagnesemia, which caused the manufacturer to place a clinical …

… guidelines have been published or actualized in the last 5 years, and we recommend
updating the existing guidelines and implementing a reliable methodology in their development. …

… Complete cloning of the Duchenne muscular dystrophy (DMD) cDNA and preliminary … -binding
domain of dystrophin associated with Duchenne muscular dystrophy, mental retardation …