Article
DMD genotypes and loss of ambulation in the CINRG Duchenne Natural History Study.
Neurology - 26 Jul 2016
Bello Luca, Morgenroth Lauren P, Gordish-Dressman Heather, Hoffman Eric P, McDonald Craig M, Cirak Sebahattin
Abstract excerpt
OBJECTIVE: To correlate time to loss of ambulation (LoA) and different truncating DMD gene mutations in a large, prospective natural history study of Duchenne muscular dystrophy (DMD), with particular attention to mutations amenable to emerging molecular treatments. METHODS: We analyzed data from the Cooperative International Neuromuscular Research Group Duchenne Natural History Study for participants with DMD...
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.
