Rafii, Michael S MD

INCLUDE Grants​

Clinical trials to prevent Alzheimer's Disease in Down Syndrome

Rafii, Michael S

AD remains one of our greatest unmet medical needs, without any approved disease-modifying therapies. Individuals with DS comprise the largest group with genetically determined AD. We propose to assess the utility of Tau PET imaging as well as CSF and blood levels of Ptau217 as outcome measures for clinical trials of AD in adults with DS.

Clinical trials to prevent Alzheimer's Disease in Down Syndrome

Rafii, Michael S

Therapies to treat AD in DS are urgently needed. This application seeks to utilize the existing expertise and experience of the Alzheimer's Clinical Trial Consortium (ACTC), Alzheimer's Biomarker Consortium for Down Syndrome (ABC-DS) and Horzion21 to establish a Trial Ready Cohort to conduct AD therapeutic trials in adults with DS in a thoughtful, ethical and scientifically-rigorous manner.

Down Syndrome Publications​