Clinical Investigation of Duchenne Muscular Dystrophy: A Methodology for Therapeutic Trials Based on Natural History Controls

Jerry R. Mendell, Michael A. Province, Richard T. Moxley, Robert C. Griggs, Michael H. Brooke, Gerald M. Fenichel, J. Philip Miller, Kenneth K. Kaiser, Wendy King, Jenny Robison, Linda Signore, Shree Pandya, Julaine M. Florence, Warren Seyfried, Stephen Mandel

Research output: Contribution to journalArticlepeer-review

51 Scopus citations

Abstract

Between 1979 and 1987 we documented the natural history of Duchenne muscular dystrophy in 170 patients, aged from 3 to 23 years, by making serial measurements in over 5000 individual evaluations. This database makes it possible to design and conduct therapeutic trials using natural history controls. Such trials do not replace the need for randomized placebo-controlled trials of promising agents but they do require fewer patients, are cost-effective, and permit the use of high-risk therapy where toxicity monitoring may be important. Natural history-controlled trials, therefore, may serve as a screening method for new therapeutic agents. Drugs showing a significant benefit can then be evaluated in a randomized controlled trial.

Original languageEnglish
Pages (from-to)808-811
Number of pages4
JournalArchives of neurology
Volume44
Issue number8
DOIs
StatePublished - Aug 1987

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