Distance to expert care for patients with lysosomal storage disorders enrolled in a real-world data research platform
One of the greatest challenges faced by patients with lysosomal storage disorders (LSDs) and their families is identifying and accessing adequate care. In this poster, we characterize the geographic distribution of centers where participants in our LSD research programs receive care, including prospective LSD centers of excellence (COEs), and describe the travel burdens associated with accessing them. A thorough understanding of patient and COE geography is essential in planning site-based clinical trials, as proper study site selection could increase the chances of successful recruitment and retention by decreasing travel burden. This poster was presented at the 18th Annual WORLDSymposium in February 2022.