The risks of therapeutic misconception and individual patient (n=1) "trials" in rare diseases such as Duchenne dystrophy.
Department of Human Genetics, Leiden University Medical Center, P.O. Box 9600, 2300 RC Leiden, The Netherlands. a.m.rus@lumc.nl
Annemieke
A
Aartsma-Rus
D002986:Clinical Trials as Topic; D006801:Humans; D020388:Muscular Dystrophy, Duchenne / Q000628:therapy*; D057285:Precision Medicine / Q000379:methods*; D055990:Therapeutic Misconception