From the study website, the purpose of this research study is “to collect blood and behavioral information from participants with Sanfilippo Syndrome Type A (MPS IIIA).”The data will help to inform future investigational treatment study into Sanfilippo.
Per the website regarding the Denali natural history study: “The expected length of the study is a screening phase and 7 study visits over approximately 102 weeks. You will have a screening phase and collection visits that will occur approximately on day 1 (baseline), week 12, week 24, week 36, week 48, week 72, and week 96. The screening phase should take approximately one hour to complete. The 7 study visits should take approximately two hours or longer to complete. The visits will include collecting and processing your samples and completing the behavioral assessment.”