Li X, Zhao L, Zhou S,et al.A comprehensive database of Duchenne and Becker muscular dystrophy patients (0–18 years old) in East China[J]. Orphanet Journal of Rare Diseases, 2015, 10: 5. Bushby K, Finkel R, Birnkrant DJ,et al.Diagnosis and management of Duchenne muscular dystrophy, ...
Li X, Zhao L, Zhou S,et al.A comprehensive database of Duchenne and Becker muscular dystrophy patients (0–18 years old) in East China[J]. Orphanet Journal of Rare Diseases, 2015, 10: 5. Bushby K, Finkel R, Birnkrant DJ,et al.Diagnosis and management of Duchenne muscular dystrophy, ...
Patients with Duchenne muscular dystrophy (DMD) show clinically relevant phenotypic variability, despite sharing the same primary biochemical defect (dystrophin deficiency). Factors contributing to this clinical variability include allelic heterogeneity
Noncoding RNAs (ncRNAs), such as miRNAs and long noncoding RNAs, are key regulators of gene expression at the post-transcriptional level and represent promising therapeutic targets and biomarkers for several human diseases, including Duchenne and Becker muscular dystrophies (DMD/BMD). A role for n...
Keywords: Duchenne/Becker muscular dystrophy (DMD/BMD); DMD gene; dystrophin; molecular diagnosis; DMD model animal; gene therapy; readthrough therapy; exon skipping therapy; antisense oligonucleotide 1. Introduction The dystrophinopathies Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (...
Resources: GeneReviews PharmGKB Table of Pharmacogenomic Biomarkers in Drug Labeling FDA Black Box Warning on GLP-1 Agonists Get ready, genetic nerds—another brand-new episode of DNA Today drops next Friday! You can always count on us to deliver fresh content every Friday. While you wait, ...