Keck School Faculty

Michael S. Rafii

Michael S. Rafii

Professor of Clinical Neurology
Medicine
ATR 9860 Mesa Rim Road Health Sciences Campus San Diego

Alzheimer’s Association : Research Champion Award, 2022

LuMind Down Syndrome Research Foundation: David Cox Award, 2016

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

The Johns Hopkins Hospital: Alpha Omega Alpha , 2006

The Johns Hopkins Hospital: Alpha Omega Alpha, 2006

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

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

Early-stage Alzheimer disease: getting trial-ready Nat Rev Neurol. 2022 07; 18(7):389-399. . View in PubMed

Development of treatments for Down syndrome Lancet Neurol. 2022 01; 21(1):22-23. . View in PubMed

Conducting clinical trials in persons with Down syndrome: summary from the NIH INCLUDE Down syndrome clinical trials readiness working group J Neurodev Disord. 2022 03 23; 14(1):22. . View in PubMed

Safety, Tolerability, and Immunogenicity of the ACI-24 Vaccine in Adults With Down Syndrome: A Phase 1b Randomized Clinical Trial JAMA Neurol. 2022 06 01; 79(6):565-574. . View in PubMed

Neurologic complications of Down syndrome: a systematic review J Neurol. 2021 Dec; 268(12):4495-4509. . View in PubMed

Alzheimer's disease associated with Down syndrome: a genetic form of dementia Lancet Neurol. 2021 11; 20(11):930-942. . View in PubMed

Increased Autoimmunity in Individuals With Down Syndrome and Moyamoya Disease Front Neurol. 2021; 12:724969. . View in PubMed

Feasibility study for detection of retinal amyloid in clinical trials: The Anti-Amyloid Treatment in Asymptomatic Alzheimer's Disease (A4) trial Alzheimers Dement (Amst). 2021; 13(1):e12199. . View in PubMed

Plasma Total-Tau and Neurofilament Light Chain as Diagnostic Biomarkers of Alzheimer's Disease Dementia and Mild Cognitive Impairment in Adults with Down Syndrome J Alzheimers Dis. 2021; 79(2):671-681. . View in PubMed

Findings of Efficacy, Safety, and Biomarker Outcomes of Atabecestat in Preclinical Alzheimer Disease: A Truncated Randomized Phase 2b/3 Clinical Trial JAMA Neurol. 2021 03 01; 78(3):293-301. . View in PubMed

Integrating Biomarker Outcomes into Clinical Trials for Alzheimer's Disease in Down Syndrome J Prev Alzheimers Dis. 2021; 8(1):48-51. . View in PubMed

The search for Alzheimer disease therapeutics - same targets, better trials? Nat Rev Neurol. 2020 11; 16(11):597-598.. View in PubMed

Alzheimer's Disease in Down Syndrome: Progress in the Design and Conduct of Drug Prevention Trials CNS Drugs. 2020 08; 34(8):785-794. . 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

Diminished Blood Pressure Profiles in Children With Down Syndrome Hypertension. 2020 03; 75(3):819-825. . 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

The AT(N) framework for Alzheimer's disease in adults with Down syndrome Alzheimers Dement (Amst). 2020; 12(1):e12062. . 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 09 01; 77(9):1099-1109. . View in PubMed

Down syndrome Nat Rev Dis Primers. 2020 02 06; 6(1):9. . View in PubMed

Effect of AZD0530 on Cerebral Metabolic Decline in Alzheimer Disease: A Randomized Clinical Trial JAMA Neurol. 2019 Oct 01; 76(10):1219-1229. . 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

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

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

The National Task Group on Intellectual Disabilities and Dementia Practices consensus recommendations for the evaluation and management of dementia in adults with intellectual disabilities Mayo Clin Proc. 2013 Aug; 88(8):831-40. . View in PubMed

Update on Alzheimer's disease therapeutics Rev Recent Clin Trials. 2013 Jun; 8(2):110-8. . 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

Functional brain imaging in the clinical assessment of consciousness PLoS Biol. 2010 Nov 30; 8(11):e1000548. . 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

Primary care screening for dementia and mild cognitive impairment JAMA. 2008 Mar 12; 299(10):1132; author reply 1133-4. . View in PubMed

Biglycan binds to alpha- and gamma-sarcoglycan and regulates their expression during development J Cell Physiol. 2006 Nov; 209(2):439-47. . View in PubMed

Case 14: a woman with bilateral Bell's palsy MedGenMed. 2006 Oct 31; 8(4):23. . View in PubMed

Compendium of cerebrovascular diseases Int Rev Psychiatry. 2006 Oct; 18(5):395-407. . View in PubMed

Biglycan regulates the expression and sarcolemmal localization of dystrobrevin, syntrophin, and nNOS FASEB J. 2006 Aug; 20(10):1724-6. . View in PubMed

Orolingual angioedema associated with ACE inhibitor use after rtPA treatment of acute stroke Neurology. 2005 Dec 27; 65(12):1906. . View in PubMed

Michael Rafii is Professor of Neurology at the Keck School of Medicine and Medical Director of the Alzheimer’s Therapeutic Research Institute (ATRI). He received his MD and PhD degrees from Brown University 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 the University of California, San Diego.

Dr. Rafii is a physician-scientist whose research focuses on developing new treatments for Alzheimer’s disease (AD) including a genetic form that occurs in people with Down syndrome (DS). He is Principal Investigator of the NIH-funded Alzheimer’s Clinical Trials Consortium - Down Syndrome (ACTC-DS), an international network of academic researchers with expertise in AD and DS.

As medical director at ATRI, he leads a team of medical and clinical monitors and provides safety oversight to ATRI’s entire portfolio of clinical trials. He serves as a scientific reviewer for the NIH and the Alzheimer's Association. His work has been featured in the New York Times, Chicago Tribune, Washington Post, Wall Street Journal and National Public Radio (NPR).

Previously, Dr. Rafii served as Medical Director of the Alzheimer’s Disease Cooperative Study, Director of the Memory Disorders Clinic, Founding Director of the Adult Down Syndrome Clinic, Director of the Comprehensive Alzheimer’s Program, Director of the Adult Neurology Residency Training program, Attending Neurologist and Associate Professor of Neurology at the University of California, San Diego.
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