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

miR-31 modulates dystrophin expression: new implications for Duchenne muscular dystrophy therapy

(2011) Davide Cacchiarelli et al.   EMBO REPORTS

Internal deletion compromises the stability of dystrophin

(2011) Davin M. Henderson et al.   HUMAN MOLECULAR GENETICS

Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study

(2011) Sebahattin Cirak et al.   LANCET

Loss of sarcolemmal nNOS is common in acquired and inherited neuromuscular disorders

(2011) E. L. Finanger Hedderick et al.   NEUROLOGY

Systemic Administration of PRO051 in Duchenne's Muscular Dystrophy

(2011) Nathalie M. Goemans et al.   NEW ENGLAND JOURNAL OF MEDICINE

Dystrophin: More than just the sum of its parts

(2010) Elisabeth Le Rumeur et al.   BIOCHIMICA ET BIOPHYSICA ACTA-PROTEINS AND PROTEOMICS

RNA-targeted splice-correction therapy for neuromuscular disease

(2010) M. J. A. Wood et al.   BRAIN

Exon-skipped dystrophins for treatment of Duchenne muscular dystrophy: Mass spectrometry mapping of most exons and cooperative domain designs based on single molecule mechanics

(2010) Christine Carag Krieger et al.   Cytoskeleton

SPP1 genotype is a determinant of disease severity in Duchenne muscular dystrophy

(2010) E. Pegoraro et al.   NEUROLOGY

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

Revertant fibres and dystrophin traces in Duchenne muscular dystrophy: Implication for clinical trials

(2010) Virginia Arechavala-Gomeza et al.   NEUROMUSCULAR DISORDERS

Best Practice Guidelines on molecular diagnostics in Duchenne/Becker muscular dystrophies

(2010) Stephen Abbs et al.   NEUROMUSCULAR DISORDERS

Our trails and trials in the subsarcolemmal cytoskeleton network and muscular dystrophy researches in the dystrophin era

(2010) Eijiro OZAWA   PROCEEDINGS OF THE JAPAN ACADEMY SERIES B-PHYSICAL AND BIOLOGICAL SCIENCES

A Renaissance for Antisense Oligonucleotide Drugs in Neurology

(2009) Toshifumi Yokota et al.   ARCHIVES OF NEUROLOGY

Analysis of Dystrophin Deletion Mutations Predicts Age of Cardiomyopathy Onset in Becker Muscular Dystrophy

(2009) Rita Wen Kaspar et al.   Circulation-Cardiovascular Genetics

DHPR α1S subunit controls skeletal muscle mass and morphogenesis

(2009) France Piétri-Rouxel et al.   EMBO JOURNAL

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

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

Dystrophins carrying spectrin-like repeats 16 and 17 anchor nNOS to the sarcolemma and enhance exercise performance in a mouse model of muscular dystrophy

(2009) Yi Lai et al.   JOURNAL OF CLINICAL INVESTIGATION

Characterization of deletion breakpoints in patients with dystrophinopathy carrying a deletion of exons 45–55 of the Duchenne muscular dystrophy (DMD) gene

(2009) Daigo Miyazaki et al.   JOURNAL OF HUMAN GENETICS

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

Immunohistological intensity measurements as a tool to assess sarcolemma-associated protein expression

(2009) V. Arechavala-Gomeza et al.   NEUROPATHOLOGY AND APPLIED NEUROBIOLOGY

Assessment of the feasibility of exon 45–55 multiexon skipping for duchenne muscular dystrophy

(2008) Laura van Vliet et al.   BMC Medical Genetics

Follow-up of three patients with a large in-frame deletion of exons 45–55 in the Duchenne muscular dystrophy (DMD) gene

(2008) Akinori Nakamura et al.   JOURNAL OF CLINICAL NEUROSCIENCE

Asymptomatic Becker muscular dystrophy in a family with a multiexon deletion

(2008) Verónica Ferreiro et al.   MUSCLE & NERVE

Sarcolemma-localized nNOS is required to maintain activity after mild exercise

(2008) Yvonne M. Kobayashi et al.   NATURE