Genotype and phenotype characterization in a large dystrophinopathic cohort with extended follow-up

Emerging genetic therapies to treat Duchenne muscular dystrophy

(2009) Stanley F Nelson et al.   CURRENT OPINION IN NEUROLOGY

Analysis of Dp71 contribution in the severity of mental retardation through comparison of Duchenne and Becker patients differing by mutation consequences on Dp71 expression

(2009) F. Daoud et al.   HUMAN MOLECULAR GENETICS

Theoretic applicability of antisense-mediated exon skipping for Duchenne muscular dystrophy mutations

(2009) Annemieke Aartsma-Rus et al.   HUMAN MUTATION

Genotype-phenotype analysis in 2,405 patients with a dystrophinopathy using the UMD-DMD database: a model of nationwide knowledgebase

(2009) Sylvie Tuffery-Giraud et al.   HUMAN MUTATION

In vivocomparison of 2′-O-methyl phosphorothioate and morpholino antisense oligonucleotides for Duchenne muscular dystrophy exon skipping

(2009) Hans A. Heemskerk et al.   JOURNAL OF GENE MEDICINE

Current understanding and management of dilated cardiomyopathy in Duchenne and Becker muscular dystrophy

(2009) Rita Wen Kaspar et al.   Journal of the American Academy of Nurse Practitioners

Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study

(2009) Maria Kinali et al.   LANCET NEUROLOGY

Characterization of the DMD/BMD patient population in Czech Republic and Slovakia using an innovative registry approach

(2009) Petr Brabec et al.   NEUROMUSCULAR DISORDERS

Clinical Heterogeneity of Duchenne Muscular Dystrophy (DMD): Definition of Sub-Phenotypes and Predictive Criteria by Long-Term Follow-Up

(2009) Isabelle Desguerre et al.   PLoS One

Mutation Analysis in a Population-Based Cohort of Boys With Duchenne or Becker Muscular Dystrophy

(2008) Christopher Cunniff et al.   JOURNAL OF CHILD NEUROLOGY

MLPA analysis/complete sequencing of the DMD gene in a group of Bulgarian Duchenne/Becker muscular dystrophy patients

(2008) Albena Todorova et al.   NEUROMUSCULAR DISORDERS

149th ENMC International Workshop and 1st TREAT-NMD Workshop on: “Planning Phase I/II Clinical trials using Systemically Delivered Antisense Oligonucleotides in Duchenne Muscular Dystrophy”

(2008) Francesco Muntoni et al.   NEUROMUSCULAR DISORDERS