Abstract
Transgene SA is developing Myodys, a non-viral plasmid gene therapy for the potential treatment of Duchenne muscular dystrophy and Becker muscular dystrophy. Phase I clinical trials have been completed, and a phase II clinical trial is planned.
MeSH terms
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Animals
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Clinical Trials as Topic
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Contraindications
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Drug Evaluation, Preclinical
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Dystrophin / genetics*
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Dystrophin / therapeutic use*
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Genetic Therapy*
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Genetic Vectors / adverse effects
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Genetic Vectors / pharmacokinetics
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Genetic Vectors / therapeutic use*
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Humans
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Mice
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Muscular Dystrophy, Duchenne / genetics*
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Muscular Dystrophy, Duchenne / therapy*
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Patents as Topic
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Plasmids / adverse effects
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Plasmids / pharmacokinetics
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Plasmids / therapeutic use*
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Structure-Activity Relationship