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Genotype and phenotype characterization in a large dystrophinopathic cohort with extended follow-up
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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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Immunohistological intensity measurements as a tool to assess sarcolemma-associated protein expression
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Differential stabilities of alternative exon-skipped rod motifs of dystrophin
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Analysis of Dystrophin Deletion Mutations Predicts Age of Cardiomyopathy Onset in Becker Muscular Dystrophy
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Dystrophins carrying spectrin-like repeats 16 and 17 anchor nNOS to the sarcolemma and enhance exercise performance in a mouse model of muscular dystrophy
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Preservation of muscle force in mdx3cv mice correlates with low-level expression of a near full-length dystrophin protein
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Local dystrophin restoration with antisense oligonucleotide PRO051
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Dystrophin levels as low as 30% are sufficient to avoid muscular dystrophy in the human
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Managing Duchenne muscular dystrophy - The additive effect of spinal surgery and home nocturnal ventilation in improving survival
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Entries in the Leiden Duchenne muscular dystrophy mutation database: An overview of mutation types and paradoxical cases that confirm the reading-frame rule
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