Clinical characterisation of Becker muscular dystrophy patients predicts favourable outcome in exon-skipping therapy

Splice Modulating Therapies for Human Disease

(2012) Pietro Spitali et al.   CELL

Dystrophin quantification and clinical correlations in Becker muscular dystrophy: implications for clinical trials

(2011) K. Anthony et al.   BRAIN

Rescue of a dystrophin-like protein by exon skipping normalizes synaptic plasticity in the hippocampus of the mdx mouse

(2011) Glenn Dallérac et al.   NEUROBIOLOGY OF DISEASE

Cardiac abnormalities in a follow-up study on carriers of Duchenne and Becker muscular dystrophy

(2011) S. M. S. van Westrum et al.   NEUROLOGY

Duchenne Muscular Dystrophy: Continuous Noninvasive Ventilatory Support Prolongs Survival

(2011) J. R. Bach et al.   Respiratory Care

Preclinical PK and PD Studies on 2′-O-Methyl-phosphorothioate RNA Antisense Oligonucleotides in the mdx Mouse Model

(2010) Hans Heemskerk et al.   MOLECULAR THERAPY

Becker muscular dystrophy patients with deletions around exon 51; a promising outlook for exon skipping therapy in Duchenne patients

(2010) A.T.J.M. Helderman-van den Enden et al.   NEUROMUSCULAR DISORDERS

Duchenne muscular dystrophy: Survival by cardio-respiratory interventions

(2010) Yuka Ishikawa et al.   NEUROMUSCULAR DISORDERS

Dose-dependent restoration of dystrophin expression in cardiac muscle of dystrophic mice by systemically delivered morpholino

(2009) B Wu et al.   GENE THERAPY

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

(2009) Annemieke Aartsma-Rus 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

A deficit of brain dystrophin impairs specific amygdala GABAergic transmission and enhances defensive behaviour in mice

(2008) Masayuki Sekiguchi et al.   BRAIN

Neuropsychiatric Disorders in Males With Duchenne Muscular Dystrophy: Frequency Rate of Attention-Deficit Hyperactivity Disorder (ADHD), Autism Spectrum Disorder, and Obsessive—Compulsive Disorder

(2008) Joseph G. M. Hendriksen et al.   JOURNAL OF CHILD NEUROLOGY

Disability and survival in Duchenne muscular dystrophy

(2008) M Kohler et al.   JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY