Ontology highlight
ABSTRACT:
SUBMITTER: Al-Zaidy S
PROVIDER: S-EPMC4254539 | biostudies-literature | 2014 Nov
REPOSITORIES: biostudies-literature
Al-Zaidy Samiah S Rodino-Klapac Louise L Mendell Jerry R JR
Pediatric neurology 20140807 5
Gene therapy for the muscular dystrophies has evolved as a promising treatment for this progressive group of disorders. Although corticosteroids and/or supportive treatments remain the standard of care for Duchenne muscular dystrophy, loss of ambulation, respiratory failure, and compromised cardiac function is the inevitable outcome. Recent developments in genetically mediated therapies have allowed for personalized treatments that strategically target individual muscular dystrophy subtypes base ...[more]