Keck School Faculty

Michael S. Rafii, MD, PhD
Michael S. Rafii, MD, PhD
Associate Professor Of Clinical of Neurology
Neurology
ATR 9860 Mesa Rim Road Health Sciences Campus San Diego
Michael Rafii is a Board-certified neurologist and Associate Professor of Clinical Neurology at the Keck School of Medicine of the University of Southern California (USC). He is also Medical Director of the Alzheimer’s Therapeutic Research Institute (ATRI) and the NIH-funded Alzheimer’s Clinical Trial Consortium (ACTC) where he oversees safety across all clinical trials. He received his MD and PhD degrees from Brown University School of Medicine and conducted neurogenetics research at Harvard Medical School. Dr. Rafii completed his residency in Neurology at the Johns Hopkins Hospital where he also served as chief resident. He then completed a fellowship in dementia at UC San Diego.

Dr. Rafii’s research focuses on the design and conduct of multi-center clinical trials for Alzheimer's disease, including a genetic form of AD which occurs in Down syndrome. He has been involved in the coordination of clinical trials, spanning phase I-III, for over 10 years. He was principal investigator of the Down Syndrome Biomarker Initiative (DSBI) and serves on the steering committee for the NIH-funded Alzheimer's Biomarker Consortium - Down syndrome (ABC-DS). Dr. Rafii is co-editor of the textbook 'Common Pathogenic Mechanisms between Down Syndrome and Alzheimer's Disease: Steps toward Therapy.’ Dr. Rafii is also 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.

Previously, 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 and Director of the 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 Clinical Trials: Moving Toward Successful Prevention CNS Drugs. 2018 Dec 17. . View in PubMed

Prevalence and Severity of Alzheimer Disease in Individuals With Down Syndrome JAMA Neurol. 2018 Nov 19. . View in PubMed

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

Diagnostic biomarkers of Alzheimer's disease in Down syndrome Lancet Neurol. 2018 Aug 29. . View in PubMed

Estimating the Evolution of Disease in the Parkinson's Progression Markers Initiative Neurodegener Dis. 2018 Aug 08; 18(4):173-190. . View in PubMed

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

Adeno-Associated Viral Vector (Serotype 2)-Nerve Growth Factor for Patients With Alzheimer Disease: A Randomized Clinical Trial JAMA Neurol. 2018 Mar 26. . 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

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

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

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

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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