Development of a real-world evidence platform for MPS Type III (Sanfilippo Syndrome)

For rare conditions such as MPS type III (Sanfilippo syndrome), it is often difficult to conduct natural history studies due to the scarcity of patients seeking care at any one clinical site. AllStripes’ real-world data (RWD) platform enables researchers to structure clinical insights without the geographic restrictions present in traditional observational studies. Here we use the example of MPS type III to demonstrate the utility of our RWD platform. This poster was presented at the 16th Annual WORLDSymposium in February 2020.