Evaluating Implementation of the Updated Care Considerations for Duchenne Muscular Dystrophy
Ong KS, Kinnett K, Soelaeman R, et al. Evaluating Implementation of the Updated Care Considerations for Duchenne Muscular Dystrophy. Pediatrics…
Duchenne muscular dystrophy awaits gene therapy
Jones D. Duchenne muscular dystrophy awaits gene therapy. Nature biotechnology 2019;37:335-337
Non-immunogenic utrophin gene therapy for the treatment of muscular dystrophy animal models
Song Y, Morales L, Malik AS, et al. Non-immunogenic utrophin gene therapy for the treatment of muscular dystrophy animal models.…
Micro-utrophin Therapy for Duchenne Muscular Dystroph
Duan D. Micro-utrophin Therapy for Duchenne Muscular Dystrophy. Molecular therapy : the journal of the American Society of Gene Therapy…
Breakpoint junction features of seven DMD deletion mutations
Keegan NP, Wilton SD, Fletcher S. Breakpoint junction features of seven DMD deletion mutations. Human genome variation 2019;6:39 Abstract…
Large in-frame 5' deletions in DMD associated with mild Duchenne muscular dystrophy: Two case reports and a review of the literature
Gibbs EM, Barthelemy F, Douine ED, et al. Large in-frame 5' deletions in DMD associated with mild Duchenne muscular dystrophy:…
Motor delays: early identification and evaluation
Noritz GH, Murphy NA. Motor delays: early identification and evaluation. Pediatrics 2013;131:e2016-27 Abstract Pediatricians often encounter children with delays…
Delayed diagnosis of Duchenne muscular dystrophy
Mohamed K, Appleton R, Nicolaides P. Delayed diagnosis of Duchenne muscular dystrophy. European journal of paediatric neurology : EJPN :…
Diagnosis of Duchenne Muscular Dystrophy in Italy in the last decade: Critical issues and areas for improvements
D'Amico A, Catteruccia M, Baranello G, et al. Diagnosis of Duchenne Muscular Dystrophy in Italy in the last decade: Critical…
Parental Reflections on the Diagnostic Process for Duchenne Muscular Dystrophy: A Qualitative Study
Bendixen RM, Houtrow A. Parental Reflections on the Diagnostic Process for Duchenne Muscular Dystrophy: A Qualitative Study. Journal of pediatric…
