TY - JOUR
T1 - Clinical Investigation of Duchenne Muscular Dystrophy
T2 - A Methodology for Therapeutic Trials Based on Natural History Controls
AU - Mendell, Jerry R.
AU - Province, Michael A.
AU - Moxley, Richard T.
AU - Griggs, Robert C.
AU - Brooke, Michael H.
AU - Fenichel, Gerald M.
AU - Miller, J. Philip
AU - Kaiser, Kenneth K.
AU - King, Wendy
AU - Robison, Jenny
AU - Signore, Linda
AU - Pandya, Shree
AU - Florence, Julaine M.
AU - Seyfried, Warren
AU - Mandel, Stephen
PY - 1987/8
Y1 - 1987/8
N2 - 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.
AB - 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.
UR - http://www.scopus.com/inward/record.url?scp=18844469600&partnerID=8YFLogxK
U2 - 10.1001/archneur.1987.00520200012009
DO - 10.1001/archneur.1987.00520200012009
M3 - Article
C2 - 3115236
AN - SCOPUS:18844469600
SN - 0003-9942
VL - 44
SP - 808
EP - 811
JO - Archives of neurology
JF - Archives of neurology
IS - 8
ER -