Exon skipping: a first in class strategy for Duchenne muscular dystrophy
Author:
Affiliation:
1. Department of Neurology, Leiden University Medical Center, Leiden, The Netherlands
2. Department of Human Genetics, Leiden University Medical Center, Leiden, The Netherlands
Funder
COST
ZonMw
Publisher
Informa UK Limited
Subject
Clinical Biochemistry,Drug Discovery,Pharmacology
Link
https://tandfonline.com/doi/pdf/10.1080/14712598.2017.1271872
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1. Evidence-based path to newborn screening for duchenne muscular dystrophy
2. Long Term Natural History Data in Ambulant Boys with Duchenne Muscular Dystrophy: 36-Month Changes
3. Individualized Prediction of Changes in 6-Minute Walk Distance for Patients with Duchenne Muscular Dystrophy
4. Association Study of Exon Variants in the NF-κB and TGFβ Pathways Identifies CD40 as a Modifier of Duchenne Muscular Dystrophy
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