Keck School Faculty

Michael S. Rafii
Michael S. Rafii
Associate Professor Of Clinical Neurology
ATR 9860 Mesa Rim Road Health Sciences Campus San Diego
Michael Rafii is a board-certified neurologist and Associate Professor of Neurology at the Keck School of Medicine of the University of Southern California (USC). He is Medical Director of the Alzheimer’s Therapeutic Research Institute (ATRI) and leads the Medical Safety Unit of the NIH-funded Alzheimer’s Clinical Trial Consortium (ACTC). \n\nDr. Rafii received his MD and PhD degrees from Brown University School of Medicine and conducted neurogenetics research at Harvard Medical School. He completed his residency in Neurology at the Johns Hopkins Hospital and fellowship in neurodegenerative diseases at UC San Diego. \n\nDr. Rafii’s research focuses on the design and conduct of multi-center clinical trials for Alzheimer's disease including a genetic form that occurs in Down syndrome. He is Principal Investigator of the NIH-funded Alzheimer’s Clinical Trial Consortium - Down Syndrome (ACTC-DS). He served as the Principal Investigator of the Down Syndrome Biomarker Initiative and the phase Ib multi-center clinical trial of the anti-amyloid vaccine ACI-24 in DS.\n\nDr. Rafii is editor of the textbook ‘Common Pathogenic Mechanisms between Down Syndrome and Alzheimer's Disease: Steps toward Therapy’ and author of the book ‘First Aid for the Neurology Boards’. He is a scientific reviewer for the National Institutes of Health and the Alzheimer's Association. He holds two patents for protein-based therapeutics which are in clinical development. Dr. Rafii’s work has been featured in the New York Times, Chicago Tribune, Wall Street Journal and on National Public Radio (NPR).\n\nPreviously, Dr. Rafii served as Medical Director of the Alzheimer’s Disease Cooperative Study (ADCS), Director of the Memory Disorders Clinic, Founding Director of the Adult Down Syndrome Clinic, Clinical Director of the Down Syndrome Center for Research and Treatment and Director of the Adult Neurology Residency Training program at UC San Diego.

LuMind Down Syndrome Research Foundation: David Cox Award, 2016

The Johns Hopkins Hospital: Alpha Omega Alpha , 2006

The Johns Hopkins Hospital: Preziosi Outstanding Neurology Resident Award, 2006

The Johns Hopkins University/Sinai Hospital: Intern of the Year, 2003

Brown University School of Medicine: Aronson Prize for Excellence in Neuroscience, 2002

Alzheimer's Disease in Down Syndrome: Progress in the Design and Conduct of Drug Prevention Trials CNS Drugs. 2020 Aug; 34(8):785-794. . View in PubMed

Safety, Efficacy, and Feasibility of Intranasal Insulin for the Treatment of Mild Cognitive Impairment and Alzheimer Disease Dementia: A Randomized Clinical Trial JAMA Neurol. 2020 Jun 22. . View in PubMed

Further understanding the connection between Alzheimer's disease and Down syndrome Alzheimers Dement. 2020 07; 16(7):1065-1077. . View in PubMed

Initiation of symptomatic medication in Alzheimer's disease clinical trials: Hypothetical versus treatment policy approach Alzheimers Dement. 2020 05; 16(5):797-803. . View in PubMed

Proteomic profiles for Alzheimer's disease and mild cognitive impairment among adults with Down syndrome spanning serum and plasma: An Alzheimer's Biomarker Consortium-Down Syndrome (ABC-DS) study Alzheimers Dement (Amst). 2020; 12(1):e12039. . View in PubMed

Effect of AZD0530 on Cerebral Metabolic Decline in Alzheimer Disease: A Randomized Clinical Trial JAMA Neurol. 2019 Jul 22. . View in PubMed

Predicting the course of Alzheimer's progression Brain Inform. 2019 Jun 28; 6(1):6. . View in PubMed

Tau PET Imaging for Staging of Alzheimer's Disease in Down Syndrome Dev Neurobiol. 2019 07; 79(7):711-715. . View in PubMed

Alzheimer's Disease Clinical Trials: Moving Toward Successful Prevention CNS Drugs. 2019 02; 33(2):99-106. . View in PubMed

Plasma Neurofilament Light and Alzheimer's Disease Biomarkers in Down Syndrome: Results from the Down Syndrome Biomarker Initiative (DSBI) J Alzheimers Dis. 2019; 70(1):131-138. . View in PubMed

Prevalence and Severity of Alzheimer Disease in Individuals With Down Syndrome JAMA Neurol. 2019 02 01; 76(2):142-143. . View in PubMed

Down syndrome Handb Clin Neurol. 2019; 167:321-336. . View in PubMed

Diagnostic biomarkers of Alzheimer's disease in Down syndrome Lancet Neurol. 2018 10; 17(10):831-832. . View in PubMed

Bayesian latent time joint mixed-effects model of progression in the Alzheimer's Disease Neuroimaging Initiative Alzheimers Dement (Amst). 2018; 10:657-668. . View in PubMed

Adeno-Associated Viral Vector (Serotype 2)-Nerve Growth Factor for Patients With Alzheimer Disease: A Randomized Clinical Trial JAMA Neurol. 2018 07 01; 75(7):834-841. . View in PubMed

Estimating the Evolution of Disease in the Parkinson's Progression Markers Initiative Neurodegener Dis. 2018; 18(4):173-190. . View in PubMed

Cerebral amyloid angiopathy in Down syndrome and sporadic and autosomal-dominant Alzheimer's disease Alzheimers Dement. 2017 Nov; 13(11):1251-1260. . View in PubMed

A phase 3 trial of IV immunoglobulin for Alzheimer disease Neurology. 2017 May 02; 88(18):1768-1775. . View in PubMed

Outcome Measures for Clinical Trials in Down Syndrome Am J Intellect Dev Disabil. 2017 05; 122(3):247-281. . View in PubMed

PET Imaging of Tau Pathology and Relationship to Amyloid, Longitudinal MRI, and Cognitive Change in Down Syndrome: Results from the Down Syndrome Biomarker Initiative (DSBI) J Alzheimers Dis. 2017; 60(2):439-450. . View in PubMed

A Randomized, Double-Blind, Placebo-Controlled, Phase II Study of Oral ELND005 (scyllo-Inositol) in Young Adults with Down Syndrome without Dementia J Alzheimers Dis. 2017; 58(2):401-411. . View in PubMed

Dissociation of Down syndrome and Alzheimer's disease effects with imaging Alzheimers Dement (N Y). 2016 Jun; 2(2):69-81. . View in PubMed

Improving Memory and Cognition in Individuals with Down Syndrome CNS Drugs. 2016 07; 30(7):567-73. . View in PubMed

Targeting tau protein in Alzheimer's disease Lancet. 2016 12 10; 388(10062):2842-2844. . View in PubMed

Advances in Alzheimer's disease drug development BMC Med. 2015 Mar 25; 13:62. . View in PubMed

The down syndrome biomarker initiative (DSBI) pilot: proof of concept for deep phenotyping of Alzheimer's disease biomarkers in down syndrome Front Behav Neurosci. 2015; 9:239. . View in PubMed

A phase1 study of stereotactic gene delivery of AAV2-NGF for Alzheimer's disease Alzheimers Dement. 2014 Sep; 10(5):571-81. . View in PubMed

Neuropsychiatric symptoms and regional neocortical atrophy in mild cognitive impairment and Alzheimer's disease Am J Alzheimers Dis Other Demen. 2014 Mar; 29(2):159-65. . View in PubMed

Preclinical Alzheimer's disease therapeutics J Alzheimers Dis. 2014; 42 Suppl 4:S545-9. . View in PubMed

Pro: Are we ready to translate Alzheimer's disease modifying therapies to people with Down syndrome? Alzheimers Res Ther. 2014; 6(5-8):60.. View in PubMed

Down's syndrome and Alzheimer's disease: towards secondary prevention Nat Rev Drug Discov. 2012 09; 11(9):655-6. . View in PubMed

Comparison of the memory performance index with standard neuropsychological measures of cognition Am J Alzheimers Dis Other Demen. 2011 05; 26(3):235-9. . View in PubMed

The pulse of drug development for Alzheimer's disease Rev Recent Clin Trials. 2010 Jan; 5(1):57-62. . View in PubMed

High-throughput, fully automated volumetry for prediction of MMSE and CDR decline in mild cognitive impairment Alzheimer Dis Assoc Disord. 2009 Apr-Jun; 23(2):139-45. . View in PubMed

Recent developments in Alzheimer's disease therapeutics BMC Med. 2009 Feb 19; 7:7. . View in PubMed

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