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New RRC, Liposomes: A practical approach
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Clinical Research
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Population frequencies of inherited neuromuscular diseases - a world survey
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Evidence-based path to newborn screening for Duchenne muscular dystrophy
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Profiles of neuromuscular diseases. Duchenne muscular dystrophy
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Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management
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Quality of life, physical disability and respiratory impairment in Duchenne muscular dystrophy
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Randomized, double-blind six-month trial of prednisone in Duchenne's muscular dystrophy
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Glucocorticoid corticosteroids for Duchenne muscular dystrophy
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Local dystrophin restoration with antisense oligonucleotide PRO051
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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
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Systemic administration of PRO051 in Duchenne's muscular dystrophy
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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
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Theoretic applicability of antisense-mediated exon skipping for Duchenne muscular dystrophy mutations
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Dystrophin and mutations: one gene, several proteins, multiple phenotypes
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The clinical, genetic and dystrophin characteristics of Becker muscular dystrophy. I. Natural history
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Comparative analysis of antisense oligonucleotide sequences for targeted skipping of exon 51 during dystrophin pre-mRNA splicing in human muscle
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Dystrophin immunity revealed by gene therapy in Duchenne muscular dystrophy
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Use of epitope libraries to identify exon-specific monoclonal antibodies for characterization of altered dystrophins in muscular dystrophy
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Immunohistological intensity measurements as a tool to assess sarcolemma-associated protein expression
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The 6-minute walk test as a new outcome measure in Duchenne muscular dystrophy
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Functional changes in Duchenne muscular dystrophy: a 12-month longitudinal cohort study
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The 6-minute walk test in Duchenne/Becker muscular dystrophy: longitudinal observations
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Therapeutics in Duchenne muscular dystrophy
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Characterization of dystrophin in muscle-biopsy specimens from patients with Duchenne's or Becker's muscular dystrophy
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X-chromosome methylation in manifesting and healthy carriers of dystrophinopathies: concordance of activation ratios among first degree female relatives and skewed inactivation as cause of the affected phenotypes
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The Effects of Low Levels of Dystrophin on Mouse Muscle Function and Pathology
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Clinical investigation in Duchenne dystrophy: 2. Determination of the ''power'' of therapeutic trials based on the natural history
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Mdx mice inducibly expressing dystrophin provide insights into the potential of gene therapy for Duchenne muscular dystrophy
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Restoring dystrophin expression in Duchenne muscular dystrophy muscle: Progress in exon skipping and stop codon read through
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Early corticosteroid treatment in 4 Duchenne muscular dystrophy patients: 14-year follow-up
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Morpholino oligomer-mediated exon skipping averts the onset of dystrophic pathology in the mdx mouse
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Efficacy of systemic morpholino exon-skipping in Duchenne dystrophy dogs
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Antisense-induced multiexon skipping for Duchenne muscular dystrophy makes more sense
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