The new frontier of genetically targeted therapies for muscle disease

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This article presents the case of a 5-year-old boy with Duchenne muscular dystrophy who is eligible to enroll in a clinical trial of gene therapy for this disorder. His parents are grappling with the decision about whether to enroll him. Among the issues under consideration are the potential risks and benefits to him, the costs of participating (because frequent, partially reimbursed travel is involved), and the potential cost savings of receiving this treatment on a research basis rather than as a clinically approved therapy. His parents seek the advice of his pediatric neurologist. After careful consideration of the various factors above, the pediatric neurologist explains to the family that participating in the trial is ethically permissible but that, given the uncertain benefits and potential for substantial expenses without benefit to the child, participation should not be regarded as ethically obligatory.

Original languageEnglish (US)
Pages (from-to)1698-1702
Number of pages5
JournalCONTINUUM Lifelong Learning in Neurology
Issue number6
StatePublished - Dec 2013
Externally publishedYes


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