Considerations to the policy of future clinical therapeutic trials in DMD.
In spite of rapidly increasing insight into the molecular basis of neuromuscular diseases, treatment still relies on convention and clinical studies. Experience with a multicentre double blind treatment study in Duchenne muscular dystrophy and with consecutive steroid treatment documentation for up to 8 years enables us to identify a series of crucial points on which to focus while planning such clinical trials. The most important seem to be: a carefully structured, detailed study, clear-cut aims and objectives, expertise of investigators, sufficient training of examiners, and careful monitoring. If patients with neuromuscular diseases are treated outside structured studies, their course should be monitored comparably. Examples of the impact of such documentation are available from the ongoing German multicentre trial on the treatment of Duchenne muscular dystrophy.
Childrens' Hospital, Johannes Gutenberg University, Mainz, Germany. reitter@kinder.klinik.uni-mainz.de
Bernd
B
Reitter
D002986:Clinical Trials as Topic / Q000592:standards / Q000639:trends; D005500:Follow-Up Studies; D006801:Humans; D015337:Multicenter Studies as Topic; D020388:Muscular Dystrophy, Duchenne / Q000628:therapy*