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Michael S. Rafii, MD, PhD

TitleAssociate Professor of Clinical Neurology
InstitutionUniversity of Southern California
DepartmentNeurology
AddressATR 9860 Mesa Rim Road
Health Sciences Campus
San Diego CA 92121
Phone+1 858 964 0638
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    Collapse Biography 
    Collapse Awards and Honors
    LuMind Down Syndrome Research Foundation2016David Cox Award
    The Johns Hopkins Hospital2006Preziosi Outstanding Neurology Resident Award
    The Johns Hopkins Hospital2006Alpha Omega Alpha
    The Johns Hopkins University/Sinai Hospital2003Intern of the Year
    Brown University School of Medicine2002Aronson Prize for Excellence in Neuroscience

    Collapse Overview 
    Collapse Overview
    Michael Rafii is Clinical Director of the Alzheimer’s Therapeutic Research Institute (ATRI) and Associate Professor of Clinical Neurology at the Keck School of Medicine of the University of Southern California (USC). He leads the ATRI Medical Safety group which provides medical oversight for all ongoing clinical trials. He received his MD and PhD degrees from Brown University School of Medicine and conducted neurogenetics research at Harvard Medical School. He holds two patents for protein-based therapeutics resulting from his work while a graduate student. Dr. Rafii completed his residency in Neurology at the Johns Hopkins Hospital where he also served as chief resident. He then undertook 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 AD clinical trials, spanning phase I-III, for over 10 years. He is principal investigator of the Down Syndrome Biomarker Initiative (DSBI) and serves on the steering committee for the Alzheimer's Biomarker Consortium in 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.' He is also author of the book, ‘First Aid for the Neurology Boards’ one of the most widely used resources by neurology residents in preparation for their board examination. He is a scientific reviewer for the National Institutes of Health and the Alzheimer's Association. Dr. Rafii is recipient of the 2016 LuMind Research Down Syndrome Foundation David Cox Award.

    Previously, Dr. Rafii served as Medical Director of the Alzheimer’s Disease Cooperative Study (ADCS), Director of the Memory Disorders Clinic and Director of the Neurology Residency Training program at UC San Diego.

    Dr. Rafii holds a joint appointment at UC San Diego.


    Collapse Research 
    Collapse Research Activities and Funding
    ACTIVE IMMUNOTHERAPY FOR COGNITIVE DECLINE IN ADULTS WITH DOWN SYNDROME
    NIH/NIA R01AG047922Aug 15, 2014 - Jun 30, 2016
    Role: Principal Investigator
    Alzheimer's Disease Cooperative Study
    NIH/NIA U19AG010483Jul 1, 1997 - Feb 28, 2018
    Role: Co-Principal Investigator

    Collapse Bibliographic 
    Collapse Publications
    Publications listed below are automatically derived from MEDLINE/PubMed and other sources, which might result in incorrect or missing publications. Researchers can login to make corrections and additions, or contact us for help.
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    1. Rafii M, Tuszynski MH, Thomas RG, Barba D, Brewer JB, Rissman RA, Siffert J, Aisen PS. Adeno-Associated Viral Vector (Serotype 2)-Nerve Growth Factor for Patients With Alzheimer Disease: A Randomized Clinical Trial. JAMA Neurol. 2018 Mar 26. PMID: 29582053.
      View in: PubMed
    2. Esbensen AJ, Hooper SR, Fidler D, Hartley SL, Edgin J, d'Ardhuy XL, Capone G, Conners FA, Mervis CB, Abbeduto L, Rafii M, Krinsky-McHale SJ, Urv T. Outcome Measures for Clinical Trials in Down Syndrome. Am J Intellect Dev Disabil. 2017 05; 122(3):247-281. PMID: 28452584.
      View in: PubMed
    3. Relkin NR, Thomas RG, Rissman RA, Brewer JB, Rafii M, van Dyck CH, Jack CR, Sano M, Knopman DS, Raman R, Szabo P, Gelmont DM, Fritsch S, Aisen PS. A phase 3 trial of IV immunoglobulin for Alzheimer disease. Neurology. 2017 May 02; 88(18):1768-1775. PMID: 28381506.
      View in: PubMed
    4. Rafii M, Lukic AS, Andrews RD, Brewer J, Rissman RA, Strother SC, Wernick MN, Pennington C, Mobley WC, Ness S, Matthews DC. 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. PMID: 28946567.
      View in: PubMed
    5. Rafii M, Skotko BG, McDonough ME, Pulsifer M, Evans C, Doran E, Muranevici G, Kesslak P, Abushakra S, Lott IT. 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. PMID: 28453471.
      View in: PubMed
    6. Rafii M. Targeting tau protein in Alzheimer's disease. Lancet. 2016 12 10; 388(10062):2842-2844. PMID: 27863808.
      View in: PubMed
    7. Rafii M. Improving Memory and Cognition in Individuals with Down Syndrome. CNS Drugs. 2016 07; 30(7):567-73. PMID: 27272473.
      View in: PubMed
    8. Rafii M, Wishnek H, Brewer JB, Donohue MC, Ness S, Mobley WC, Aisen PS, Rissman RA. 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. PMID: 26441570; PMCID: PMC4568340.
    9. Rafii M. Pro: Are we ready to translate Alzheimer's disease modifying therapies to people with Down syndrome? Alzheimers Res Ther. 2014; 6(5-8):60. PMID: 25478025; PMCID: PMC4255529.
    10. Rafii M, Baumann TL, Bakay RA, Ostrove JM, Siffert J, Fleisher AS, Herzog CD, Barba D, Pay M, Salmon DP, Chu Y, Kordower JH, Bishop K, Keator D, Potkin S, Bartus RT. A phase1 study of stereotactic gene delivery of AAV2-NGF for Alzheimer's disease. Alzheimers Dement. 2014 Sep; 10(5):571-81. PMID: 24411134.
      View in: PubMed
    11. Rafii M. Preclinical Alzheimer's disease therapeutics. J Alzheimers Dis. 2014; 42 Suppl 4:S545-9. PMID: 25079804.
      View in: PubMed
    12. Rafii M, Taylor CS, Kim HT, Desikan RS, Fleisher AS, Katibian D, Brewer JB, Dale AM, Aisen PS. 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. PMID: 24164929.
      View in: PubMed
    13. Rafii M, Taylor C, Coutinho A, Kim K, Galasko D. Comparison of the memory performance index with standard neuropsychological measures of cognition. Am J Alzheimers Dis Other Demen. 2011 05; 26(3):235-9. PMID: 21406427; PMCID: PMC3568924.
    14. Rafii M. The pulse of drug development for Alzheimer's disease. Rev Recent Clin Trials. 2010 Jan; 5(1):57-62. PMID: 20205688.
      View in: PubMed
    15. Kovacevic S, Rafii M, Brewer JB. 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. PMID: 19474571; PMCID: PMC2688740.
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