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Michael S. Rafii, MD, PhD

TitleAssociate Professor Of Clinical of 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 Medical 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 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. 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 Alzheimer's Biomarker Consortium - Down syndrome (ABC-DS) as well as the American Academy of Developmental Medicine National Task Group on Intellectual Disabilities and Dementia Practices. Dr. Rafii is co-editor of the textbook 'Common Pathogenic Mechanisms between Down Syndrome and Alzheimer's Disease: Steps toward Therapy.’ 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.


    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 MS, Santoro SL. Prevalence and Severity of Alzheimer Disease in Individuals With Down Syndrome. JAMA Neurol. 2018 Nov 19. PMID: 30452497.
      View in: PubMed
    2. Li D, Iddi S, Thompson WK, Rafii MS, Aisen PS, Donohue MC. Bayesian latent time joint mixed-effects model of progression in the Alzheimer's Disease Neuroimaging Initiative. Alzheimers Dement (Amst). 2018; 10:657-668. PMID: 30456292.
      View in: PubMed
    3. Rafii MS. Diagnostic biomarkers of Alzheimer's disease in Down syndrome. Lancet Neurol. 2018 Aug 29. PMID: 30172623.
      View in: PubMed
    4. Iddi S, Li D, Aisen PS, Rafii MS, Litvan I, Thompson WK, Donohue MC. Estimating the Evolution of Disease in the Parkinson's Progression Markers Initiative. Neurodegener Dis. 2018 Aug 08; 18(4):173-190. PMID: 30089306.
      View in: PubMed
    5. Rafii MS, 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
    6. Esbensen AJ, Hooper SR, Fidler D, Hartley SL, Edgin J, d'Ardhuy XL, Capone G, Conners FA, Mervis CB, Abbeduto L, Rafii MS, 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
    7. Carmona-Iragui M, Balasa M, Benejam B, Alcolea D, Fernández S, Videla L, Sala I, Sánchez-Saudinós MB, Morenas-Rodriguez E, Ribosa-Nogué R, Illán-Gala I, Gonzalez-Ortiz S, Clarimón J, Schmitt F, Powell DK, Bosch B, Lladó A, Rafii MS, Head E, Molinuevo JL, Blesa R, Videla S, Lleó A, Sánchez-Valle R, Fortea J. Cerebral amyloid angiopathy in Down syndrome and sporadic and autosomal-dominant Alzheimer's disease. Alzheimers Dement. 2017 Nov; 13(11):1251-1260. PMID: 28463681.
      View in: PubMed
    8. Relkin NR, Thomas RG, Rissman RA, Brewer JB, Rafii MS, 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
    9. Rafii MS, 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
    10. Rafii MS, 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
    11. Rafii MS. Targeting tau protein in Alzheimer's disease. Lancet. 2016 12 10; 388(10062):2842-2844. PMID: 27863808.
      View in: PubMed
    12. Rafii MS. Improving Memory and Cognition in Individuals with Down Syndrome. CNS Drugs. 2016 07; 30(7):567-73. PMID: 27272473.
      View in: PubMed
    13. Matthews DC, Lukic AS, Andrews RD, Marendic B, Brewer J, Rissman RA, Mosconi L, Strother SC, Wernick MN, Mobley WC, Ness S, Schmidt ME, Rafii MS. Dissociation of Down syndrome and Alzheimer's disease effects with imaging. Alzheimers Dement (N Y). 2016 Jun; 2(2):69-81. PMID: 28642933.
      View in: PubMed
    14. Rafii MS, 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.
    15. Rafii MS. 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.
    16. Rafii MS, 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
    17. Rafii MS. Preclinical Alzheimer's disease therapeutics. J Alzheimers Dis. 2014; 42 Suppl 4:S545-9. PMID: 25079804.
      View in: PubMed
    18. Rafii MS, 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
    19. Ness S, Rafii M, Aisen P, Krams M, Silverman W, Manji H. Down's syndrome and Alzheimer's disease: towards secondary prevention. Nat Rev Drug Discov. 2012 09; 11(9):655-6. PMID: 22935789.
      View in: PubMed
    20. Rafii MS, 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.
    21. Rafii MS. The pulse of drug development for Alzheimer's disease. Rev Recent Clin Trials. 2010 Jan; 5(1):57-62. PMID: 20205688.
      View in: PubMed
    22. Kovacevic S, Rafii MS, 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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