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The New Frontier of Genetically Targeted Therapies for Muscle Disease

Kang, Peter, B.

doi: 10.1212/01.CON.0000440666.79792.84
Ethical Perspectives

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.

Address correspondence to Dr Peter B. Kang, Division of Pediatric Neurology, University of Florida College of Medicine, PO Box 100296, Gainesville, FL 32610,

Relationship Disclosure: Dr Kang has served as an officer and trustee of the Massachusetts Medical Society and received grant funding from Isis Pharmaceuticals, Inc; the Muscular Dystrophy Association; and the NIH. Dr Kang has received honoraria from the AAN, the American College of Medical Genetics, Boston Children’s Hospital, Brooks Publishing, the Fondazione Cariplo, the Massachusetts Medical Society, the US Department of Health and Human Services-Health Resources and Services Administration’s National Vaccine Injury Compensation Program, and the US Department of Veterans Affairs.

Unlabeled Use of Products/Investigational Use Disclosure: Dr Kang discusses therapies for the treatment of muscle disease, all of which are unlabeled.

© 2013 American Academy of Neurology
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