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Michael S. Rafii, MD, PhD

Title(s)Associate Professor Of Clinical Neurology
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 Neurology at the Keck School of Medicine. He received his MD and PhD degrees from Brown University and conducted neurogenetics research at Harvard Medical School. He completed his Neurology residency 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 clinical trials for Alzheimer's disease including a genetic form that occurs in people with Down syndrome. He is Principal Investigator of the Alzheimer’s Clinical Trial Consortium - Down Syndrome (ACTC-DS). Dr. Rafii is editor of the textbook ‘Common Pathogenic Mechanisms between Down Syndrome and Alzheimer's Disease: Steps toward Therapy’ and 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. He holds two patents for therapeutics which are in clinical development. 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, Director of the Memory Disorders Clinic, Founding Director of the Adult Down Syndrome Clinic, Director of the Comprehensive Alzheimer’s Program and Director of the Adult Neurology Residency Training program at the University of California, San Diego.

    Collapse Research 
    Collapse Research Activities and Funding
    Clinical trials to prevent Alzheimer's Disease in Down Syndrome
    NIH R61AG066543Sep 15, 2019 - Aug 31, 2021
    Role: Principal Investigator
    Alzheimer's Disease Cooperative Study
    NIH U19AG010483May 1, 2015 - Nov 30, 2017
    Role: Co-Principal Investigator
    ACTIVE IMMUNOTHERAPY FOR COGNITIVE DECLINE IN ADULTS WITH DOWN SYNDROME
    NIH R01AG047922Aug 15, 2014 - Jun 30, 2019
    Role: 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. to make corrections and additions.
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    Altmetrics Details PMC Citations indicate the number of times the publication was cited by articles in PubMed Central, and the Altmetric score represents citations in news articles and social media. (Note that publications are often cited in additional ways that are not shown here.) Fields are based on how the National Library of Medicine (NLM) classifies the publication's journal and might not represent the specific topic of the publication. Translation tags are based on the publication type and the MeSH terms NLM assigns to the publication. Some publications (especially newer ones and publications not in PubMed) might not yet be assigned Field or Translation tags.) Click a Field or Translation tag to filter the publications.
    1. Findings of Efficacy, Safety, and Biomarker Outcomes of Atabecestat in Preclinical Alzheimer Disease: A Truncated Randomized Phase 2b/3 Clinical Trial. JAMA Neurol. 2021 Jan 19. Sperling R, Henley D, Aisen PS, Raman R, Donohue MC, Ernstrom K, Rafii MS, Streffer J, Shi Y, Karcher K, Raghavan N, Tymofyeyev Y, Bogert J, Brashear HR, Novak G, Thipphawong J, Saad ZS, Kolb H, Rofael H, Sanga P, Romano G. PMID: 33464300.
      View in: PubMed   Mentions:    Fields:    
    2. Integrating Biomarker Outcomes into Clinical Trials for Alzheimer's Disease in Down Syndrome. J Prev Alzheimers Dis. 2021; 8(1):48-51. Rafii MS, Zaman S, Handen BL. PMID: 33336224.
      View in: PubMed   Mentions:    Fields:    
    3. 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. 2020 Dec 16. Petersen ME, Rafii MS, Zhang F, Hall J, Julovich D, Ances BM, Schupf N, Krinsky-McHale SJ, Mapstone M, Silverman W, Lott I, Klunk W, Head E, Christian B, Foroud T, Lai F, Diana Rosas H, Zaman S, Wang MC, Tycko B, Lee J, Handen B, Hartley S, Fortea J, O'Bryant S. PMID: 33337378.
      View in: PubMed   Mentions:    Fields:    
    4. The search for Alzheimer disease therapeutics - same targets, better trials? Nat Rev Neurol. 2020 Nov; 16(11):597-598. Rafii MS, Aisen PS. PMID: 32963375.
      View in: PubMed   Mentions:    Fields:    
    5. The AT(N) framework for Alzheimer's disease in adults with Down syndrome. Alzheimers Dement (Amst). 2020; 12(1):e12062. PMID: 33134477.
      View in: PubMed   Mentions:
    6. Neurologic complications of Down syndrome: a systematic review. J Neurol. 2020 Sep 12. Santoro JD, Pagarkar D, Chu DT, Rosso M, Paulsen KC, Levitt P, Rafii MS. PMID: 32920658.
      View in: PubMed   Mentions:    Fields:    
    7. 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 Sep 01; 77(9):1099-1109. Craft S, Raman R, Chow TW, Rafii MS, Sun CK, Rissman RA, Donohue MC, Brewer JB, Jenkins C, Harless K, Gessert D, Aisen PS. PMID: 32568367.
      View in: PubMed   Mentions: 6     Fields:    
    8. Alzheimer's Disease in Down Syndrome: Progress in the Design and Conduct of Drug Prevention Trials. CNS Drugs. 2020 08; 34(8):785-794. Rafii MS. PMID: 32506291.
      View in: PubMed   Mentions:    Fields:    
    9. 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. Petersen ME, Zhang F, Schupf N, Krinsky-McHale SJ, Hall J, Mapstone M, Cheema A, Silverman W, Lott I, Rafii MS, Handen B, Klunk W, Head E, Christian B, Foroud T, Lai F, Rosas HD, Zaman S, Ances BM, Wang MC, Tycko B, Lee JH, O'Bryant S. PMID: 32626817.
      View in: PubMed   Mentions:
    10. Further understanding the connection between Alzheimer's disease and Down syndrome. Alzheimers Dement. 2020 07; 16(7):1065-1077. Snyder HM, Bain LJ, Brickman AM, Carrillo MC, Esbensen AJ, Espinosa JM, Fernandez F, Fortea J, Hartley SL, Head E, Hendrix J, Kishnani PS, Lai F, Lao P, Lemere C, Mobley W, Mufson EJ, Potter H, Zaman SH, Granholm AC, Rosas HD, Strydom A, Whitten MS, Rafii MS. PMID: 32544310.
      View in: PubMed   Mentions:    Fields:    
    11. Initiation of symptomatic medication in Alzheimer's disease clinical trials: Hypothetical versus treatment policy approach. Alzheimers Dement. 2020 05; 16(5):797-803. Donohue MC, Model F, Delmar P, Volye N, Liu-Seifert H, Rafii MS, Aisen PS. PMID: 32270600.
      View in: PubMed   Mentions:    Fields:    
    12. Effect of AZD0530 on Cerebral Metabolic Decline in Alzheimer Disease: A Randomized Clinical Trial. JAMA Neurol. 2019 Jul 22. 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. PMID: 31329216.
      View in: PubMed   Mentions: 6     Fields:    
    13. Predicting the course of Alzheimer's progression. Brain Inform. 2019 Jun 28; 6(1):6. Iddi S, Li D, Aisen PS, Rafii MS, Thompson WK, Donohue MC. PMID: 31254120.
      View in: PubMed   Mentions:
    14. Alzheimer's Disease Clinical Trials: Moving Toward Successful Prevention. CNS Drugs. 2019 02; 33(2):99-106. Rafii MS, Aisen PS. PMID: 30560544.
      View in: PubMed   Mentions: 5     Fields:    Translation:Humans
    15. Prevalence and Severity of Alzheimer Disease in Individuals With Down Syndrome. JAMA Neurol. 2019 02 01; 76(2):142-143. Rafii MS, Santoro SL. PMID: 30452497.
      View in: PubMed   Mentions: 2     Fields:    Translation:Humans
    16. 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. Rafii MS, Donohue MC, Matthews DC, Muranevici G, Ness S, O'Bryant SE, Rissman RA. PMID: 31156181.
      View in: PubMed   Mentions: 3     Fields:    Translation:Humans
    17. Down syndrome. Handb Clin Neurol. 2019; 167:321-336. Rafii MS, Kleschevnikov AM, Sawa M, Mobley WC. PMID: 31753140.
      View in: PubMed   Mentions:    Fields:    Translation:Humans
    18. Tau PET Imaging for Staging of Alzheimer's Disease in Down Syndrome. Dev Neurobiol. 2019 07; 79(7):711-715. Rafii MS. PMID: 30536948.
      View in: PubMed   Mentions: 4     Fields:    Translation:HumansAnimals
    19. Bayesian latent time joint mixed-effects model of progression in the Alzheimer's Disease Neuroimaging Initiative. Alzheimers Dement (Amst). 2018; 10:657-668. Li D, Iddi S, Thompson WK, Rafii MS, Aisen PS, Donohue MC. PMID: 30456292.
      View in: PubMed   Mentions:
    20. Diagnostic biomarkers of Alzheimer's disease in Down syndrome. Lancet Neurol. 2018 10; 17(10):831-832. Rafii MS. PMID: 30172623.
      View in: PubMed   Mentions:    Fields:    Translation:Humans
    21. Estimating the Evolution of Disease in the Parkinson's Progression Markers Initiative. Neurodegener Dis. 2018; 18(4):173-190. Iddi S, Li D, Aisen PS, Rafii MS, Litvan I, Thompson WK, Donohue MC. PMID: 30089306.
      View in: PubMed   Mentions: 1     Fields:    Translation:Humans
    22. 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. Rafii MS, Tuszynski MH, Thomas RG, Barba D, Brewer JB, Rissman RA, Siffert J, Aisen PS. PMID: 29582053.
      View in: PubMed   Mentions: 20     Fields:    Translation:HumansCells
    23. Outcome Measures for Clinical Trials in Down Syndrome. Am J Intellect Dev Disabil. 2017 05; 122(3):247-281. PMID: 28452584.
      View in: PubMed   Mentions: 9     Fields:    Translation:Humans
    24. 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   Mentions: 13     Fields:    Translation:Humans
    25. A phase 3 trial of IV immunoglobulin for Alzheimer disease. Neurology. 2017 May 02; 88(18):1768-1775. PMID: 28381506.
      View in: PubMed   Mentions: 23     Fields:    Translation:HumansCTClinical Trials
    26. 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   Mentions: 16     Fields:    Translation:Humans
    27. 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   Mentions: 4     Fields:    Translation:HumansCTClinical Trials
    28. Targeting tau protein in Alzheimer's disease. Lancet. 2016 12 10; 388(10062):2842-2844. PMID: 27863808.
      View in: PubMed   Mentions: 3     Fields:    Translation:Humans
    29. Improving Memory and Cognition in Individuals with Down Syndrome. CNS Drugs. 2016 07; 30(7):567-73. PMID: 27272473.
      View in: PubMed   Mentions: 1     Fields:    Translation:HumansAnimals
    30. Dissociation of Down syndrome and Alzheimer's disease effects with imaging. Alzheimers Dement (N Y). 2016 Jun; 2(2):69-81. 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. PMID: 28642933.
      View in: PubMed   Mentions:
    31. 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   Mentions:
    32. Advances in Alzheimer's disease drug development. BMC Med. 2015 Mar 25; 13:62. PMID: 25857341.
      View in: PubMed   Mentions: 23     Fields:    Translation:Humans
    33. 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   Mentions: 2     Fields:    
    34. 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   Mentions: 45     Fields:    Translation:HumansCellsCTClinical Trials
    35. Preclinical Alzheimer's disease therapeutics. J Alzheimers Dis. 2014; 42 Suppl 4:S545-9. PMID: 25079804.
      View in: PubMed   Mentions: 3     Fields:    Translation:HumansAnimals
    36. 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   Mentions: 9     Fields:    Translation:Humans
    37. 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   Mentions: 21     Fields:    Translation:HumansPHPublic Health
    38. 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   Mentions: 3     Fields:    Translation:HumansPHPublic Health
    39. The pulse of drug development for Alzheimer's disease. Rev Recent Clin Trials. 2010 Jan; 5(1):57-62. Rafii MS. PMID: 20205688.
      View in: PubMed   Mentions: 3     Fields:    Translation:HumansAnimalsPHPublic Health
    40. 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. Kovacevic S, Rafii MS, Brewer JB. PMID: 19474571.
      View in: PubMed   Mentions: 42     Fields:    Translation:Humans
    41. Recent developments in Alzheimer's disease therapeutics. BMC Med. 2009 Feb 19; 7:7. Rafii MS, Aisen PS. PMID: 19228370.
      View in: PubMed   Mentions: 52     Fields:    Translation:Humans
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