Very Low Residual Dystrophin Quantity Is Associated with Milder Dystrophinopathy

de Féraudy, Yvan; Ben Yaou, Rabah; Wahbi, Karim; Stalens, Caroline; Stantzou, Amalia; Laugel, Vincent; Desguerre, Isabelle; the FILNEMUS Network, for; Servais, Laurent; Leturcq, France; Amthor, Helge

doi:10.1002/ana.25951

Public

Very Low Residual Dystrophin Quantity Is Associated with Milder Dystrophinopathy

Published in Annals of Neurology • Nov 7, 2020

Authors:

Yvan de Féraudy

Rabah Ben Yaou

Karim Wahbi

Abstract

OBJECTIVE: This study was undertaken to determine whether a low residual quantity of dystrophin protein is associated with delayed clinical milestones in patients with DMD mutations. METHODS: We performed a retrospective multicentric cohort study by using molecular and clinical data from patients wi...

Subject

Dystrophin

Duchenne muscular dystrophy

Medicine

Research Assistant

AI chat, annotations, notes & similar papers

Finding related papers...

Discussions

(0)

No comments yet

Be the first to share your thoughts!