28/10/2020
As part of the DevelopAKUre consortium, PSR was responsible for the project management, regulatory set-up, development of study documents, site set-up, site management, monitoring, data management, and study logistics (central labs, central evaluations, a shipment of samples, etc.). In very close collaboration with the AKU Society, we were able to enroll 140 patients from all over Europe, and beyond, at the three participating study sites (in Liverpool, Paris, and Piešťany) in less than a year. Despite the fact that this was a 4-year study with a high burden for the patients, as well as a design in which 50% of the patients did not receive treatment, we only had a limited number of drop-outs.
This study has been featured in the most recent issue of Rare Revolution Magazine
https://www.rarerevolutionmagazine.com/blog/ground-breaking-patient-led-aku-study-published-in-the-lancet #
And you can also read our complete case study here https://bit.ly/3mPQbo6PSRalkaptonuria
A digital magazine giving a voice to patients affected by RARE conditions and the charities that represent and support them.