Denali sponsoring observational research study (natural history) for Sanfilippo Type A

August 25, 2022

Denali Therapeutics has provided information about their observational research study natural history) for children with Sanfilippo Syndrome Type A.

Below is a flyer provided by Denali. More information and sign-up is available here.

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.”

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