ObjectiveTo clarify the current state of methodology of clinical trials for rare neurological diseases in children, and to provide a basis for the further optimization of the trial design. MethodsData
In rare diseases, there are many unanswered questions that are critical to clinical development. Among them, one important question is how to choose primary endpoints that translate into meaningful im
Additional file 4: Supplementary Fig. 4 Maximum extent of individual expenses considered acceptable for participation in a clinical trial. Graphical presentation of part four of the survey, asking for
The primary objective of the study is to determine the effect of pozelimab on active CD55-deficient protein-losing enteropathy (PLE; CHAPLE).The secondary objectives of the study are:* To evaluate the
