Michael S. Rafii, MD, PhD

Title(s)Associate Professor Of Clinical Neurology
SchoolKeck School of Medicine of Usc
AddressATR 9860 Mesa Rim Road
Health Sciences Campus
San Diego CA 92121
Phone+1 858 964 0638
vCardDownload vCard

    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 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’s research focuses on developing new treatments for Alzheimer’s disease, including a genetic form that occurs in people with Down syndrome. 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 Down syndrome.

    Dr. Rafii also directs ATRI’s Medical Safety Unit, which is comprised of 35 staff members including 4 physicians and provides medical and safety oversight to ATRI’s entire portfolio of clinical trials. Dr. Rafii 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, 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 and Associate Professor of Neurology at the University of California, San Diego.

    Collapse Research 
    Collapse Research Activities and Funding
    Precision Medicine for Inflammatory Treatment for Alzheimer's Disease in Down Syndrome
    NIH R01AG073979Sep 30, 2021 - Aug 31, 2024
    Role: Co-Principal Investigator
    Clinical trials to prevent Alzheimer's Disease in Down Syndrome
    NIH R61AG066543Sep 15, 2019 - Aug 31, 2021
    Role: Principal Investigator
    ACTIVE IMMUNOTHERAPY FOR COGNITIVE DECLINE IN ADULTS WITH DOWN SYNDROME
    NIH R01AG047922Aug 15, 2014 - Jun 30, 2019
    Role: Principal Investigator
    Alzheimer's Disease Cooperative Study
    NIH U19AG010483Jul 1, 1997 - Feb 28, 2021
    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. to make corrections and additions.
    Newest   |   Oldest   |   Most Cited   |   Most Discussed   |   Timeline   |   Field Summary   |   Plain Text
    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. Alzheimer's disease associated with Down syndrome: a genetic form of dementia. Lancet Neurol. 2021 Nov; 20(11):930-942. Fortea J, Zaman SH, Hartley S, Rafii MS, Head E, Carmona-Iragui M. PMID: 34687637.
      View in: PubMed   Mentions:
    2. 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. Ngolab J, Donohue M, Belsha A, Salazar J, Cohen P, Jaiswal S, Tan V, Gessert D, Korouri S, Aggarwal NT, Alber J, Johnson K, Jicha G, van Dyck C, Lah J, Salloway S, Sperling RA, Aisen PS, Rafii MS, Rissman RA. PMID: 34430703.
      View in: PubMed   Mentions: 1  
    3. Findings of Efficacy, Safety, and Biomarker Outcomes of Atabecestat in Preclinical Alzheimer Disease: A Truncated Randomized Phase 2b/3 Clinical Trial. JAMA Neurol. 2021 Mar 01; 78(3):293-301. 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: 5     Fields:    
    4. 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: 2     Fields:    Translation:Humans
    5. 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. 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 JH, Handen B, Hartley S, Fortea J, O'Bryant S, Alzheimer’s Biomarker Consortium –Down Syndrome (ABC-DS) . PMID: 33337378.
      View in: PubMed   Mentions: 5     Fields:    Translation:Humans
    6. 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: 3     Fields:    
    7. The AT(N) framework for Alzheimer's disease in adults with Down syndrome. Alzheimers Dement (Amst). 2020; 12(1):e12062. Rafii MS, Ances BM, Schupf N, Krinsky-McHale SJ, Mapstone M, Silverman W, Lott I, Klunk W, Head E, Christian B, Lai F, Rosas HD, Zaman S, Petersen ME, Strydom A, Fortea J, Handen B, O'Bryant S. PMID: 33134477.
      View in: PubMed   Mentions: 4  
    8. 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: 2     Fields:    
    9. 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. 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: 27     Fields:    Translation:Humans
    10. 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:    Translation:HumansAnimals
    11. 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, Alzheimer's Biomarker Consortium – Down Syndrome (ABC-DS) . PMID: 32626817.
      View in: PubMed   Mentions: 6  
    12. 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: 6     Fields:    Translation:Humans
    13. 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, Alzheimer's Disease Neuroimaging Initiative . PMID: 32270600.
      View in: PubMed   Mentions:    Fields:    
    14. Effect of AZD0530 on Cerebral Metabolic Decline in Alzheimer Disease: A Randomized Clinical Trial. JAMA Neurol. 2019 Oct 01; 76(10):1219-1229. 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: 25     Fields:    
    15. 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, Alzheimer’s Disease Neuroimaging Initiative . PMID: 31254120.
      View in: PubMed   Mentions: 4  
    16. 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: 9     Fields:    Translation:Humans
    17. 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: 5     Fields:    Translation:Humans
    18. 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: 9     Fields:    Translation:Humans
    19. Down syndrome. Handb Clin Neurol. 2019; 167:321-336. Rafii MS, Kleschevnikov AM, Sawa M, Mobley WC. PMID: 31753140.
      View in: PubMed   Mentions: 4     Fields:    Translation:Humans
    20. 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: 6     Fields:    Translation:HumansAnimals
    21. 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
    22. 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: 6  
    23. 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: 5     Fields:    Translation:Humans
    24. 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, AAV2-NGF Study Team . PMID: 29582053.
      View in: PubMed   Mentions: 43     Fields:    Translation:HumansCells
    25. Outcome Measures for Clinical Trials in Down Syndrome. Am J Intellect Dev Disabil. 2017 05; 122(3):247-281. 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 Working Group . PMID: 28452584.
      View in: PubMed   Mentions: 24     Fields:    Translation:Humans
    26. Cerebral amyloid angiopathy in Down syndrome and sporadic and autosomal-dominant Alzheimer's disease. Alzheimers Dement. 2017 Nov; 13(11):1251-1260. 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. PMID: 28463681; PMCID: PMC5660938.
      View in: PubMed   Mentions: 24     Fields:    Translation:Humans
    27. A phase 3 trial of IV immunoglobulin for Alzheimer disease. Neurology. 2017 May 02; 88(18):1768-1775. 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, Alzheimer's Disease Cooperative Study . PMID: 28381506.
      View in: PubMed   Mentions: 31     Fields:    Translation:HumansCTClinical Trials
    28. 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. Rafii MS, Lukic AS, Andrews RD, Brewer J, Rissman RA, Strother SC, Wernick MN, Pennington C, Mobley WC, Ness S, Matthews DC, Down Syndrome Biomarker Initiative and the Alzheimer’s Disease Neuroimaging Initiative . PMID: 28946567.
      View in: PubMed   Mentions: 33     Fields:    Translation:Humans
    29. 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. Rafii MS, Skotko BG, McDonough ME, Pulsifer M, Evans C, Doran E, Muranevici G, Kesslak P, Abushakra S, Lott IT, ELND005-DS Study Group . PMID: 28453471; PMCID: PMC5777855.
      View in: PubMed   Mentions: 9     Fields:    Translation:HumansCTClinical Trials
    30. Targeting tau protein in Alzheimer's disease. Lancet. 2016 12 10; 388(10062):2842-2844. Rafii MS. PMID: 27863808.
      View in: PubMed   Mentions: 4     Fields:    Translation:Humans
    31. Improving Memory and Cognition in Individuals with Down Syndrome. CNS Drugs. 2016 07; 30(7):567-73. Rafii MS, Rafii MS. PMID: 27272473.
      View in: PubMed   Mentions: 2     Fields:    Translation:HumansAnimals
    32. 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: 19  
    33. 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. Rafii MS, Wishnek H, Brewer JB, Donohue MC, Ness S, Mobley WC, Aisen PS, Rissman RA, Rafii MS, Wishnek H, Brewer JB, Donohue MC, Ness S, Mobley WC, Aisen PS, Rissman RA. PMID: 26441570; PMCID: PMC4568340.
      View in: PubMed   Mentions: 37  
    34. Advances in Alzheimer's disease drug development. BMC Med. 2015 Mar 25; 13:62. Rafii MS, Aisen PS, Rafii MS, Aisen PS. PMID: 25857341; PMCID: PMC4373002.
      View in: PubMed   Mentions: 26     Fields:    Translation:Humans
    35. Pro: Are we ready to translate Alzheimer's disease modifying therapies to people with Down syndrome? Alzheimers Res Ther. 2014; 6(5-8):60. Rafii MS, Rafii MS. PMID: 25478025; PMCID: PMC4255529.
      View in: PubMed   Mentions: 2     Fields:    
    36. A phase1 study of stereotactic gene delivery of AAV2-NGF for Alzheimer's disease. Alzheimers Dement. 2014 Sep; 10(5):571-81. 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, 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. PMID: 24411134.
      View in: PubMed   Mentions: 58     Fields:    Translation:HumansCellsCTClinical Trials
    37. Preclinical Alzheimer's disease therapeutics. J Alzheimers Dis. 2014; 42 Suppl 4:S545-9. Rafii MS, Rafii MS. PMID: 25079804.
      View in: PubMed   Mentions: 4     Fields:    Translation:HumansAnimals
    38. 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. Hanna RF, Ward TJ, Chow DS, Lagana SM, Moreira RK, Emond JC, Weintraub JL, Prince MR, Rafii MS, Taylor CS, Kim HT, Desikan RS, Fleisher AS, Katibian D, Brewer JB, Dale AM, Aisen PS. PMID: 24164929; PMCID: PMC5351414.
      View in: PubMed   Mentions: 10     Fields:    Translation:Humans
    39. Update on Alzheimer's disease therapeutics. Rev Recent Clin Trials. 2013 Jun; 8(2):110-8. Rafii MS, Rafii MS. PMID: 23859061.
      View in: PubMed   Mentions: 4     Fields:    Translation:Humans
    40. Down's syndrome and Alzheimer's disease: towards secondary prevention. Nat Rev Drug Discov. 2012 09; 11(9):655-6. Ness S, Rafii M, Aisen P, Krams M, Silverman W, Manji H, Ness S, Rafii M, Aisen P, Krams M, Silverman W, Manji H. PMID: 22935789.
      View in: PubMed   Mentions: 21     Fields:    Translation:HumansPHPublic Health
    41. Comparison of the memory performance index with standard neuropsychological measures of cognition. Am J Alzheimers Dis Other Demen. 2011 05; 26(3):235-9. Rafii MS, Taylor C, Coutinho A, Kim K, Galasko D, Rafii MS, Taylor C, Coutinho A, Kim K, Galasko D. PMID: 21406427; PMCID: PMC3568924.
      View in: PubMed   Mentions: 3     Fields:    Translation:HumansPHPublic Health
    42. The pulse of drug development for Alzheimer's disease. Rev Recent Clin Trials. 2010 Jan; 5(1):57-62. Rafii MS, Rafii MS. PMID: 20205688.
      View in: PubMed   Mentions: 3     Fields:    Translation:HumansAnimalsPHPublic Health
    43. 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, Alzheimer's Disease Neuroimaging Initiative , Kovacevic S, Rafii MS, Brewer JB, Alzheimer's Disease Neuroimaging Initiative. PMID: 19474571; PMCID: PMC2688740.
      View in: PubMed   Mentions: 51     Fields:    Translation:Humans
    44. Recent developments in Alzheimer's disease therapeutics. BMC Med. 2009 Feb 19; 7:7. Rafii MS, Aisen PS, Rafii MS, Aisen PS. PMID: 19228370; PMCID: PMC2649159.
      View in: PubMed   Mentions: 59     Fields:    Translation:Humans
    Michael's Networks
    Concepts (123)
    Derived automatically from this person's publications.
    _
    Co-Authors (24)
    People in Profiles who have published with this person.
    _
    Similar People (60)
    People who share similar concepts with this person.
    _
    Same Department
    Search Department
    _