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Michael S. Rafii, MD, PhD

Professor of Clinical Neurology

Associate Director of ACTC (Alzheimer's Clinical Trial Consortium) and Director of Medical Safety

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Phone +1 858 964 0638
Email mrafii@usc.edu
CTSI Profile

Publications

  • Relationship between use of anti-platelet agents, oral anti-coagulants, and Aß burden with cerebral microhemorrhages in cognitively asymptomatic adults Alzheimers Dement. 2025 May; 21(5):e70167. . View in PubMed
  • Differences in cholinergic terminal density in adults with Down syndrome compared to neurotypical controls measured by [18F]-fluoroethoxybenzovesamicol positron emission tomography imaging Neurobiol Aging. 2025 Apr; 148:50-60. . View in PubMed
  • Brain cholinergic terminal density utilizing [18F]-fluoroethoxybenzovesamicol PET in adults with Down’s syndrome: Relationship to amyloid PET and cognitive performance Alzheimers Dement. 2025 Apr; 21(4):e70134. . View in PubMed
  • Reflecting on progress, envisioning the future for research in Down syndrome Lancet. 2025 Mar 20. . View in PubMed
  • A head-to-head comparison of multiple amyloid PET radiotracers for Down syndrome clinical trials medRxiv. 2025 Mar 19. . View in PubMed
  • Clinical and neuropathological analysis of Down syndrome over 7 decades of life J Neuropathol Exp Neurol. 2025 Feb 01; 84(2):168-173. . View in PubMed
  • The mediating role of plasma glial fibrillary acidic protein in amyloid and tau pathology in Down’s syndrome Alzheimers Dement. 2025 Jan; 21(1):e14359. . View in PubMed
  • Artificial intelligence-enabled safety monitoring in Alzheimer’s disease clinical trials J Prev Alzheimers Dis. 2025 Jan; 12(1):100002. . View in PubMed
  • Diagnostic abnormalities, disease severity and immunotherapy responsiveness in individuals with Down syndrome regression disorder Sci Rep. 2024 12 28; 14(1):30865. . View in PubMed
  • Effects of the therapeutic correction of U1 snRNP complex on Alzheimer’s disease Sci Rep. 2024 12 03; 14(1):30085. . View in PubMed
  • The amyloid clock: mapping Alzheimer’s disease in Down syndrome Lancet Neurol. 2024 Dec; 23(12):1173-1174. . View in PubMed
  • Age-Related Changes in the Cholinergic System in Adults with Down Syndrome Assessed Using [18F]-Fluoroethoxybenzovesamicol Positron Emission Tomography Imaging medRxiv. 2024 Nov 01. . View in PubMed
  • The Alzheimer’s Disease Neuroimaging Initiative Clinical Core Alzheimers Dement. 2024 Oct; 20(10):7361-7368. . View in PubMed
  • De novo variants in immune regulatory genes in Down syndrome regression disorder J Neurol. 2024 Aug; 271(8):5567-5576. . View in PubMed
  • Assessing amyloid PET positivity and cognitive function in Down syndrome to guide clinical trials targeting amyloid Alzheimers Dement. 2024 08; 20(8):5570-5577. . View in PubMed
  • Revised criteria for diagnosis and staging of Alzheimer’s disease: Alzheimer’s Association Workgroup Alzheimers Dement. 2024 08; 20(8):5143-5169. . View in PubMed
  • Revised criteria for the diagnosis and staging of Alzheimer’s disease Nat Med. 2024 Aug; 30(8):2121-2124. . View in PubMed
  • Addressing challenges in health care and research for people with Down syndrome Lancet. 2024 05 11; 403(10439):1830-1833. . View in PubMed
  • Safety and tolerability of intravenous immunoglobulin infusion in Down syndrome regression disorder Am J Med Genet A. 2024 05; 194(5):e63524. . View in PubMed
  • Comparison of tau spread in people with Down syndrome versus autosomal-dominant Alzheimer’s disease: a cross-sectional study Lancet Neurol. 2024 May; 23(5):500-510. . View in PubMed
  • From understanding to action: Exploring molecular connections of Down syndrome to Alzheimer’s disease for targeted therapeutic approach Alzheimers Dement (Amst). 2024 Apr-Jun; 16(2):e12580. . View in PubMed
  • Neuroimaging abnormalities associated with immunotherapy responsiveness in Down syndrome regression disorder Ann Clin Transl Neurol. 2024 Apr; 11(4):1034-1045. . View in PubMed
  • Plasma Aß42/Aß40 and phospho-tau217 concentration ratios increase the accuracy of amyloid PET classification in preclinical Alzheimer’s disease Alzheimers Dement. 2024 02; 20(2):1214-1224. . View in PubMed
  • Down Syndrome in a New Era for Alzheimer Disease JAMA. 2023 12 12; 330(22):2157-2158. . View in PubMed
  • Hypovitaminosis D in persons with Down syndrome and autism spectrum disorder J Neurodev Disord. 2023 10 25; 15(1):35. . View in PubMed
  • Trial of Solanezumab in Preclinical Alzheimer’s Disease N Engl J Med. 2023 Sep 21; 389(12):1096-1107. . View in PubMed
  • Alternative Diagnoses in the Work Up of Down Syndrome Regression Disorder J Autism Dev Disord. 2023 Aug 16. . View in PubMed
  • Immunotherapy responsiveness and risk of relapse in Down syndrome regression disorder Transl Psychiatry. 2023 08 08; 13(1):276. . View in PubMed
  • Amyloid-Lowering Monoclonal Antibodies for the Treatment of Early Alzheimer’s Disease CNS Drugs. 2023 08; 37(8):671-677. . View in PubMed
  • Adverse childhood experiences and the development of Down syndrome regression disorder Am J Med Genet A. 2023 07; 191(7):1769-1782. . View in PubMed
  • Detection and treatment of Alzheimer’s disease in its preclinical stage Nat Aging. 2023 05; 3(5):520-531. . View in PubMed
  • Safety and tolerability of lumbar puncture for the evaluation of Alzheimer’s disease Alzheimers Dement (Amst). 2023 Apr-Jun; 15(2):e12431. . View in PubMed
  • The AHEAD 3-45 Study: Design of a prevention trial for Alzheimer’s disease Alzheimers Dement. 2023 04; 19(4):1227-1233. . View in PubMed
  • Immunotherapy Responsiveness and Risk of Relapse in Down Syndrome Regression Disorder Res Sq. 2023 Feb 13. . View in PubMed
  • Editorial: Appropriate Use Recommendations for Lecanemab J Prev Alzheimers Dis. 2023; 10(3):356. . View in PubMed
  • Comparison of amyloid burden in individuals with Down syndrome versus autosomal dominant Alzheimer’s disease: a cross-sectional study Lancet Neurol. 2023 01; 22(1):55-65. . View in PubMed
  • Early-stage Alzheimer disease: getting trial-ready Nat Rev Neurol. 2022 07; 18(7):389-399. . View in PubMed
  • Evidence of neuroinflammation and immunotherapy responsiveness in individuals with down syndrome regression disorder J Neurodev Disord. 2022 06 03; 14(1):35. . View in PubMed
  • Safety, Tolerability, and Immunogenicity of the ACI-24 Vaccine in Adults With Down Syndrome: A Phase 1b Randomized Clinical Trial JAMA Neurol. 2022 06 01; 79(6):565-574. . View in PubMed
  • Conducting clinical trials in persons with Down syndrome: summary from the NIH INCLUDE Down syndrome clinical trials readiness working group J Neurodev Disord. 2022 03 23; 14(1):22. . View in PubMed
  • Development of treatments for Down syndrome Lancet Neurol. 2022 01; 21(1):22-23. . View in PubMed
  • Assessment and Diagnosis of Down Syndrome Regression Disorder: International Expert Consensus Front Neurol. 2022; 13:940175. . View in PubMed
  • Neurologic complications of Down syndrome: a systematic review J Neurol. 2021 Dec; 268(12):4495-4509. . View in PubMed
  • Alzheimer’s disease associated with Down syndrome: a genetic form of dementia Lancet Neurol. 2021 11; 20(11):930-942. . View in PubMed
  • Findings of Efficacy, Safety, and Biomarker Outcomes of Atabecestat in Preclinical Alzheimer Disease: A Truncated Randomized Phase 2b/3 Clinical Trial JAMA Neurol. 2021 03 01; 78(3):293-301. . View in PubMed
  • 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. . View in PubMed
  • Integrating Biomarker Outcomes into Clinical Trials for Alzheimer’s Disease in Down Syndrome J Prev Alzheimers Dis. 2021; 8(1):48-51. . View in PubMed
  • 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. . View in PubMed
  • Increased Autoimmunity in Individuals With Down Syndrome and Moyamoya Disease Front Neurol. 2021; 12:724969. . View in PubMed
  • The search for Alzheimer disease therapeutics – same targets, better trials? Nat Rev Neurol. 2020 11; 16(11):597-598.. View in PubMed
  • 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. . View in PubMed
  • Alzheimer’s Disease in Down Syndrome: Progress in the Design and Conduct of Drug Prevention Trials CNS Drugs. 2020 08; 34(8):785-794. . View in PubMed
  • Further understanding the connection between Alzheimer’s disease and Down syndrome Alzheimers Dement. 2020 07; 16(7):1065-1077. . View in PubMed
  • Initiation of symptomatic medication in Alzheimer’s disease clinical trials: Hypothetical versus treatment policy approach Alzheimers Dement. 2020 05; 16(5):797-803. . View in PubMed
  • Diminished Blood Pressure Profiles in Children With Down Syndrome Hypertension. 2020 03; 75(3):819-825. . View in PubMed
  • Down syndrome Nat Rev Dis Primers. 2020 02 06; 6(1):9. . View in PubMed
  • 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. . View in PubMed
  • The AT(N) framework for Alzheimer’s disease in adults with Down syndrome Alzheimers Dement (Amst). 2020; 12(1):e12062. . View in PubMed
  • Effect of AZD0530 on Cerebral Metabolic Decline in Alzheimer Disease: A Randomized Clinical Trial JAMA Neurol. 2019 Oct 01; 76(10):1219-1229. . View in PubMed
  • Tau PET Imaging for Staging of Alzheimer’s Disease in Down Syndrome Dev Neurobiol. 2019 07; 79(7):711-715. . View in PubMed
  • Predicting the course of Alzheimer’s progression Brain Inform. 2019 Jun 28; 6(1):6. . View in PubMed
  • Prevalence and Severity of Alzheimer Disease in Individuals With Down Syndrome JAMA Neurol. 2019 02 01; 76(2):142-143. . View in PubMed
  • Alzheimer’s Disease Clinical Trials: Moving Toward Successful Prevention CNS Drugs. 2019 02; 33(2):99-106. . View in PubMed
  • Down syndrome Handb Clin Neurol. 2019; 167:321-336. . View in PubMed
  • 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. . View in PubMed
  • Diagnostic biomarkers of Alzheimer’s disease in Down syndrome Lancet Neurol. 2018 10; 17(10):831-832. . View in PubMed
  • 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. . View in PubMed
  • Estimating the Evolution of Disease in the Parkinson’s Progression Markers Initiative Neurodegener Dis. 2018; 18(4):173-190. . View in PubMed
  • Bayesian latent time joint mixed-effects model of progression in the Alzheimer’s Disease Neuroimaging Initiative Alzheimers Dement (Amst). 2018; 10:657-668. . View in PubMed
  • Cerebral amyloid angiopathy in Down syndrome and sporadic and autosomal-dominant Alzheimer’s disease Alzheimers Dement. 2017 Nov; 13(11):1251-1260. . View in PubMed
  • A phase 3 trial of IV immunoglobulin for Alzheimer disease Neurology. 2017 May 02; 88(18):1768-1775. . View in PubMed
  • Outcome Measures for Clinical Trials in Down Syndrome Am J Intellect Dev Disabil. 2017 05; 122(3):247-281. . View in PubMed
  • 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. . View in PubMed
  • 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. . View in PubMed
  • Targeting tau protein in Alzheimer’s disease Lancet. 2016 12 10; 388(10062):2842-2844. . View in PubMed
  • Improving Memory and Cognition in Individuals with Down Syndrome CNS Drugs. 2016 07; 30(7):567-73. . View in PubMed
  • Dissociation of Down syndrome and Alzheimer’s disease effects with imaging Alzheimers Dement (N Y). 2016 Jun; 2(2):69-81. . View in PubMed
  • Advances in Alzheimer’s disease drug development BMC Med. 2015 Mar 25; 13:62. . View in PubMed
  • 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. . View in PubMed
  • A phase1 study of stereotactic gene delivery of AAV2-NGF for Alzheimer’s disease Alzheimers Dement. 2014 Sep; 10(5):571-81. . View in PubMed
  • 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. . View in PubMed
  • Preclinical Alzheimer’s disease therapeutics J Alzheimers Dis. 2014; 42 Suppl 4:S545-9. . View in PubMed
  • Pro: Are we ready to translate Alzheimer’s disease modifying therapies to people with Down syndrome? Alzheimers Res Ther. 2014; 6(5-8):60.. View in PubMed
  • The National Task Group on Intellectual Disabilities and Dementia Practices consensus recommendations for the evaluation and management of dementia in adults with intellectual disabilities Mayo Clin Proc. 2013 Aug; 88(8):831-40. . View in PubMed
  • Update on Alzheimer’s disease therapeutics Rev Recent Clin Trials. 2013 Jun; 8(2):110-8. . View in PubMed
  • Down’s syndrome and Alzheimer’s disease: towards secondary prevention Nat Rev Drug Discov. 2012 09; 11(9):655-6. . View in PubMed
  • Comparison of the memory performance index with standard neuropsychological measures of cognition Am J Alzheimers Dis Other Demen. 2011 05; 26(3):235-9. . View in PubMed
  • Functional brain imaging in the clinical assessment of consciousness PLoS Biol. 2010 Nov 30; 8(11):e1000548. . View in PubMed
  • The pulse of drug development for Alzheimer’s disease Rev Recent Clin Trials. 2010 Jan; 5(1):57-62. . View in PubMed
  • 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. . View in PubMed
  • Recent developments in Alzheimer’s disease therapeutics BMC Med. 2009 Feb 19; 7:7. . View in PubMed
  • Primary care screening for dementia and mild cognitive impairment JAMA. 2008 Mar 12; 299(10):1132; author reply 1133-4. . View in PubMed
  • Biglycan binds to alpha- and gamma-sarcoglycan and regulates their expression during development J Cell Physiol. 2006 Nov; 209(2):439-47. . View in PubMed
  • Case 14: a woman with bilateral Bell’s palsy MedGenMed. 2006 Oct 31; 8(4):23. . View in PubMed
  • Compendium of cerebrovascular diseases Int Rev Psychiatry. 2006 Oct; 18(5):395-407. . View in PubMed
  • Biglycan regulates the expression and sarcolemmal localization of dystrobrevin, syntrophin, and nNOS FASEB J. 2006 Aug; 20(10):1724-6. . View in PubMed
  • Orolingual angioedema associated with ACE inhibitor use after rtPA treatment of acute stroke Neurology. 2005 Dec 27; 65(12):1906. . View in PubMed

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