A major challenge for rare disease clinical trials is the limited amount of available information for making robust statistical inference. While external data presents information integration opportun
Number (percent) of orphan medicines categorised according to total number of individuals studied for 6 and 12 months (long term) prior to marketing authorisation.
The data shared in the Zenodo repository include clinical and instrumental data from a randomized clinical trial open label phase II in Friedreich's Ataxia patients that underwent a 6 months treatment
BackgroundIn clinical research, the most appropriate way to assess the effect of an intervention is to conduct a randomized controlled trial (RCT). In the field of rare diseases, conducting an RCT is