title: Risks in a Trial of an Innovative Treatment of Duchenne Muscular Dystrophy.
abstract: Studies of innovative therapies for muscular dystrophy raise unique ethical issues. The disease is currently untreatable and relentlessly progressive. A number of potentially efficacious treatments are being developed, but like all treatments, they may have unforeseen adverse effects. Nevertheless, patients and families, facing a bleak future, may be willing to take the gamble and try the treatments. Many doctors are eager to study them. But should institutional review boards approve them? This article discusses these issues and recounts the ways that one such study elicited different responses from different institutional review boards.
authors.affiliation: Department of Medical Ethics and Philosophy of Medicine, Erasmus University Medical Center, Rotterdam, Netherlands;
authors.forename: Wendy
authors.initials: W
authors.lastname: Bos
authors.affiliation: Department of Paediatrics, Leiden University Medical Center, Leiden, Netherlands;
authors.forename: Anna E
authors.initials: AE
authors.lastname: Westra
authors.affiliation: Faculty of Medicine and Life Sciences, Hasselt University, Hasselt, Belgium; and.
authors.forename: Wim
authors.initials: W
authors.lastname: Pinxten
authors.affiliation: Children's Mercy Hospital, Kansas City, Missouri.
authors.forename: Matthew P
authors.initials: MP
authors.lastname: Mayer
authors.affiliation: Children's Mercy Hospital, Kansas City, Missouri jlantos@cmh.edu.
authors.forename: John D
authors.initials: JD
authors.lastname: Lantos
mesh_terms: D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D002986:Clinical Trials as Topic / Q000941:ethics* / Q000331:legislation & jurisprudence; D064420:Drug-Related Side Effects and Adverse Reactions / Q000517:prevention & control*; D017041:Ethics Committees; D032984:Ethics, Research; D006801:Humans; D020388:Muscular Dystrophy, Duchenne / Q000628:therapy*; D061214:Patient Safety / Q000331:legislation & jurisprudence* / Q000592:standards; D012306:Risk; D016896:Treatment Outcome