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Michael S. Rafii, MD, PhD

Title(s)Associate Professor Of Clinical Neurology
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 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 Director of the Medical Safety Unit at the Alzheimer’s Therapeutic Research Institute (ATRI) and the NIH-funded Alzheimer’s Clinical Trial Consortium (ACTC) where he leads a team of 4 physicians and 21 clinical monitors who oversee safety across all clinical trials. He received his MD and PhD (neuroscience) degrees from Brown University School of Medicine and conducted neurogenetics research at Harvard Medical School. Dr. Rafii completed his Neurology residency 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 (phase I-III) for Alzheimer's disease, including a genetic form of AD which occurs in Down syndrome. He is principal investigator of the NIH-funded Alzheimer’s Clinical Trial Consortium for Down Syndrome (ACTC-DS) and serves on the steering committee for the Alzheimer's Biomarker Consortium for Down Syndrome (ABC-DS). Dr. Rafii is editor of the textbook 'Common Pathogenic Mechanisms between Down Syndrome and Alzheimer's Disease: Steps toward Therapy’ as well as author of ‘First Aid for the Neurology Boards.’ He is a scientific reviewer for the National Institutes of Health and the Alzheimer's Association. Dr. Rafii’s work has been featured in the New York Times, Chicago Tribune, Wall Street Journal and on National Public Radio (NPR).

    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 Adult Neurology Residency Training program at UC San Diego.

    Collapse Research 
    Collapse Research Activities and Funding
    Clinical trials to prevent Alzheimer's Disease in Down Syndrome
    NIH/NIA R61AG066543Sep 15, 2019 - Aug 31, 2021
    Role: Principal Investigator
    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 29, 2020
    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.
    List All   |   Timeline
    1. van Dyck CH, Nygaard HB, Chen K, Donohue MC, Raman R, Rissman RA, Brewer JB, Koeppe RA, Chow TW, Rafii MS, Gessert D, Choi J, Turner RS, Kaye JA, Gale SA, Reiman EM, Aisen PS, Strittmatter SM. Effect of AZD0530 on Cerebral Metabolic Decline in Alzheimer Disease: A Randomized Clinical Trial. JAMA Neurol. 2019 Jul 22. PMID: 31329216.
      View in: PubMed
    2. Iddi S, Li D, Aisen PS, Rafii MS, Thompson WK, Donohue MC. Predicting the course of Alzheimer's progression. Brain Inform. 2019 Jun 28; 6(1):6. PMID: 31254120.
      View in: PubMed
    3. Rafii MS, Aisen PS. Alzheimer's Disease Clinical Trials: Moving Toward Successful Prevention. CNS Drugs. 2019 Feb; 33(2):99-106. PMID: 30560544.
      View in: PubMed
    4. Rafii MS, Santoro SL. Prevalence and Severity of Alzheimer Disease in Individuals With Down Syndrome. JAMA Neurol. 2019 Feb 01; 76(2):142-143. PMID: 30452497.
      View in: PubMed
    5. Rafii MS, Donohue MC, Matthews DC, Muranevici G, Ness S, O'Bryant SE, Rissman RA. 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. PMID: 31156181.
      View in: PubMed
    6. Rafii MS, Kleschevnikov AM, Sawa M, Mobley WC. Down syndrome. Handb Clin Neurol. 2019; 167:321-336. PMID: 31753140.
      View in: PubMed
    7. Rafii MS. Tau PET Imaging for Staging of Alzheimer's Disease in Down Syndrome. Dev Neurobiol. 2019 Jul; 79(7):711-715. PMID: 30536948.
      View in: PubMed
    8. Rafii MS. Diagnostic biomarkers of Alzheimer's disease in Down syndrome. Lancet Neurol. 2018 10; 17(10):831-832. PMID: 30172623.
      View in: PubMed
    9. 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
    10. 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; 18(4):173-190. PMID: 30089306.
      View in: PubMed
    11. 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 07 01; 75(7):834-841. PMID: 29582053.
      View in: PubMed
    12. 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
    13. 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
    14. 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
    15. 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
    16. 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
    17. Rafii MS. Targeting tau protein in Alzheimer's disease. Lancet. 2016 12 10; 388(10062):2842-2844. PMID: 27863808.
      View in: PubMed
    18. Rafii MS. Improving Memory and Cognition in Individuals with Down Syndrome. CNS Drugs. 2016 07; 30(7):567-73. PMID: 27272473.
      View in: PubMed
    19. 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
    20. 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.
      View in: PubMed
    21. Rafii MS, Aisen PS. Advances in Alzheimer's disease drug development. BMC Med. 2015 Mar 25; 13:62. PMID: 25857341.
      View in: PubMed
    22. 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.
      View in: PubMed
    23. 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
    24. Rafii MS. Preclinical Alzheimer's disease therapeutics. J Alzheimers Dis. 2014; 42 Suppl 4:S545-9. PMID: 25079804.
      View in: PubMed
    25. 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
    26. 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
    27. 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.
      View in: PubMed
    28. 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
    29. 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.
      View in: PubMed
    30. Rafii MS, Aisen PS. Recent developments in Alzheimer's disease therapeutics. BMC Med. 2009 Feb 19; 7:7. PMID: 19228370.
      View in: PubMed