{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,9]],"date-time":"2026-09-09T10:55:55Z","timestamp":1788951355859,"version":"build-2803163510"},"reference-count":57,"publisher":"Springer Science and Business Media LLC","issue":"1","license":[{"start":{"date-parts":[[2020,10,12]],"date-time":"2020-10-12T00:00:00Z","timestamp":1602460800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"},{"start":{"date-parts":[[2020,10,12]],"date-time":"2020-10-12T00:00:00Z","timestamp":1602460800000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"}],"funder":[{"DOI":"10.13039\/100013425","name":"Trinity College of Arts and Sciences, Duke University","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100013425","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["Life Sci Soc Policy"],"published-print":{"date-parts":[[2020,12]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:p>In the United States alone, the prevalence of AD is expected to more than double from six million people in 2019 to nearly 14 million people in 2050. Meanwhile, the track record for developing treatments for AD has been marked by decades of failure. But recent progress in genetics, neuroscience and gene editing suggest that effective treatments could be on the horizon. The arrival of such treatments would have profound implications for the way we diagnose, triage, study, and allocate resources to Alzheimer\u2019s patients. Because the disease is not rare and because it strikes late in life, the development of therapies that are expensive and efficacious but less than cures, will pose particular challenges to healthcare infrastructure. We have a window of time during which we can begin to anticipate just, equitable and salutary ways to accommodate a disease-modifying therapy Alzheimer\u2019s disease. Here we consider the implications for caregivers, clinicians, researchers, and the US healthcare system of the availability of an expensive, presymptomatic treatment for a common late-onset neurodegenerative disease for which diagnosis can be difficult.<\/jats:p>","DOI":"10.1186\/s40504-020-00106-2","type":"journal-article","created":{"date-parts":[[2020,10,11]],"date-time":"2020-10-11T23:02:36Z","timestamp":1602457356000},"update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":8,"title":["Good problems to have? Policy and societal implications of a disease-modifying therapy for presymptomatic late-onset Alzheimer\u2019s disease"],"prefix":"10.1186","volume":"16","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-1076-1423","authenticated-orcid":false,"given":"Misha","family":"Angrist","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anna","family":"Yang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Boris","family":"Kantor","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ornit","family":"Chiba-Falek","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2020,10,12]]},"reference":[{"key":"106_CR1","doi-asserted-by":"publisher","unstructured":"Alzheimer's Association. 2020. 2020 Alzheimer's disease facts and figures. Alzheimer's and Dementia. 16(3):391\u2013 460. https:\/\/doi.org\/10.1002\/alz.12068","DOI":"10.1002\/alz.12068"},{"key":"106_CR2","doi-asserted-by":"publisher","first-page":"1192","DOI":"10.1038\/nbt.3084","volume":"32","author":"M Abou-El-Enein","year":"2014","unstructured":"Abou-El-Enein, M., G. Bauer, and P. Reinke. 2014. The business case for cell and gene therapies. Nature Biotechnology. 32: 1192. https:\/\/doi.org\/10.1038\/nbt.3084.","journal-title":"Nature Biotechnology."},{"key":"106_CR3","doi-asserted-by":"publisher","first-page":"179","DOI":"10.1002\/ppul.24203","volume":"54","author":"S Al-Zaidy","year":"2019","unstructured":"Al-Zaidy, S., A.S. Pickard, K. Kotha, et al. 2019. Health outcomes in spinal muscular atrophy type 1 following AVXS-101 gene replacement therapy. Pediatric Pulmonology 54: 179\u2013185. https:\/\/doi.org\/10.1002\/ppul.24203.","journal-title":"Pediatric Pulmonology"},{"key":"106_CR4","doi-asserted-by":"publisher","first-page":"329","DOI":"10.1038\/s41586-018-0087-1","volume":"557","author":"RA Barker","year":"2018","unstructured":"Barker, R.A., M. Gotz, and M. Parmar. 2018. New approaches for brain repair-from rescue to reprogramming. Nature. 557: 329\u2013334. https:\/\/doi.org\/10.1038\/s41586-018-0087-1.","journal-title":"Nature."},{"key":"106_CR5","doi-asserted-by":"publisher","first-page":"431","DOI":"10.1053\/j.gastro.2018.10.024","volume":"156","author":"TF Baumert","year":"2019","unstructured":"Baumert, T.F., T. Berg, J.K. Lim, et al. 2019. Status of direct-acting antiviral therapy for Hepatitis C virus infection and remaining challenges. Gastroenterology. 156: 431\u2013445. https:\/\/doi.org\/10.1053\/j.gastro.2018.10.024.","journal-title":"Gastroenterology."},{"key":"106_CR6","series-title":"STAT+","volume-title":"The maddening saga of how an Alzheimer's 'cabal' thwarted progress toward a cure for decades","author":"S Begley","year":"2019","unstructured":"Begley S. The maddening saga of how an Alzheimer's 'cabal' thwarted progress toward a cure for decades. STAT+. 2019; https:\/\/www.statnews.com\/2019\/06\/25\/alzheimers-cabal-thwarted-progress-toward-cure\/."},{"key":"106_CR7","doi-asserted-by":"publisher","unstructured":"Brown, E.E., S. Kumar, T.K. Rajji, et al. 2020. Anticipating and mitigating the impact of the COVID-19 pandemic on Alzheimer's disease and related dementias. The American Journal of Geriatric Psychiatry. https:\/\/doi.org\/10.1016\/j.jagp.2020.04.010.","DOI":"10.1016\/j.jagp.2020.04.010"},{"key":"106_CR8","doi-asserted-by":"publisher","first-page":"3252","DOI":"10.1093\/brain\/awx272","volume":"140","author":"FJ Bustos","year":"2017","unstructured":"Bustos, F.J., E. Ampuero, N. Jury, et al. 2017. Epigenetic editing of the Dlg4\/PSD95 gene improves cognition in aged and Alzheimer's disease mice. Brain. 140: 3252\u20133268. https:\/\/doi.org\/10.1093\/brain\/awx272.","journal-title":"Brain."},{"key":"106_CR9","doi-asserted-by":"publisher","first-page":"1302","DOI":"10.1126\/science.342.6164.1302","volume":"342","author":"J Cohen","year":"2013","unstructured":"Cohen, J. 2013. Pharmaceuticals. Advocates protest the cost of a hepatitis C cure. Science. 342: 1302\u20131303. https:\/\/doi.org\/10.1126\/science.342.6164.1302.","journal-title":"Science."},{"key":"106_CR10","doi-asserted-by":"publisher","first-page":"S21","DOI":"10.1016\/S0960-9776(15)30015-1","volume":"24","author":"CD Corneliussen-James","year":"2015","unstructured":"Corneliussen-James, C.D. 2015. IN02 metavivor: Fighting prejudice. The Breast. 24: S21.","journal-title":"The Breast."},{"key":"106_CR11","doi-asserted-by":"publisher","first-page":"110","DOI":"10.3389\/fnmol.2019.00110","volume":"12","author":"A Cota-Coronado","year":"2019","unstructured":"Cota-Coronado, A., N.F. Diaz-Martinez, E. Padilla-Camberos, et al. 2019. Editing the central nervous system through CRISPR\/Cas9 systems. Frontiers in Molecular Neuroscience 12: 110. https:\/\/doi.org\/10.3389\/fnmol.2019.00110.","journal-title":"Frontiers in Molecular Neuroscience"},{"key":"106_CR12","doi-asserted-by":"publisher","first-page":"470","DOI":"10.1212\/WNL.0b013e318294b1cf","volume":"81","author":"TM Dall","year":"2013","unstructured":"Dall, T.M., M.V. Storm, R. Chakrabarti, et al. 2013. Supply and demand analysis of the current and future US neurology workforce. Neurology. 81: 470\u2013478. https:\/\/doi.org\/10.1212\/WNL.0b013e318294b1cf.","journal-title":"Neurology."},{"key":"106_CR13","doi-asserted-by":"publisher","first-page":"949","DOI":"10.1016\/j.drudis.2019.01.019","volume":"24","author":"JJ Darrow","year":"2019","unstructured":"Darrow, J.J. 2019. Luxturna: FDA documents reveal the value of a costly gene therapy. Drug Discovery Today 24: 949\u2013954. https:\/\/doi.org\/10.1016\/j.drudis.2019.01.019.","journal-title":"Drug Discovery Today"},{"key":"106_CR14","unstructured":"Deslatte, M. 2019. Louisiana reaches 'Netflix-model' deal to tackle hepatitis C. New York: Associated Press. https:\/\/apnews.com\/bc074b5c06024926a5c58163de8bab9d. Accessed 11 Sept 2020."},{"key":"106_CR15","doi-asserted-by":"publisher","first-page":"175","DOI":"10.1186\/s12916-018-1162-z","volume":"16","author":"CH Douglass","year":"2018","unstructured":"Douglass, C.H., A. Pedrana, J.V. Lazarus, et al. 2018. Pathways to ensure universal and affordable access to hepatitis C treatment. BMC Medicine 16: 175. https:\/\/doi.org\/10.1186\/s12916-018-1162-z.","journal-title":"BMC Medicine"},{"key":"106_CR16","doi-asserted-by":"publisher","first-page":"661","DOI":"10.1016\/j.jval.2019.03.012","volume":"22","author":"MF Drummond","year":"2019","unstructured":"Drummond, M.F., P.J. Neumann, S.D. Sullivan, et al. 2019. Analytic considerations in applying a general economic evaluation reference case to gene therapy. Value in Health 22: 661\u2013668. https:\/\/doi.org\/10.1016\/j.jval.2019.03.012.","journal-title":"Value in Health"},{"key":"106_CR17","doi-asserted-by":"publisher","first-page":"388","DOI":"10.1016\/S1474-4422(18)30123-6","volume":"17","author":"C Dufouil","year":"2018","unstructured":"Dufouil, C., and M.M. Glymour. 2018. Prediction to prevention in Alzheimer's disease and dementia. Lancet Neurology 17: 388\u2013389. https:\/\/doi.org\/10.1016\/S1474-4422(18)30123-6.","journal-title":"Lancet Neurology"},{"key":"106_CR18","doi-asserted-by":"publisher","first-page":"1256","DOI":"10.1038\/s41591-020-0938-9","volume":"26","author":"S Dujardin","year":"2020","unstructured":"Dujardin, S., C. Commins, A. Lathuiliere, et al. 2020. Tau molecular diversity contributes to clinical heterogeneity in Alzheimer's disease. Nature Medicine 26: 1256\u20131263. https:\/\/doi.org\/10.1038\/s41591-020-0938-9.","journal-title":"Nature Medicine"},{"key":"106_CR19","doi-asserted-by":"publisher","unstructured":"El-Hayek, Y.H., R.E. Wiley, C.P. Khoury, et al. 2019. Tip of the iceberg: Assessing the global socioeconomic costs of Alzheimer's disease and related dementias and strategic implications for stakeholders. Journal of Alzheimer's Disease. https:\/\/doi.org\/10.3233\/JAD-190426.","DOI":"10.3233\/JAD-190426"},{"key":"106_CR20","volume-title":"Bluebird's gene therapy for a rare blood disease will cost $1.8 million. Cue the pricing debate","author":"A Feuerstein","year":"2019","unstructured":"Feuerstein, A. 2019. Bluebird's gene therapy for a rare blood disease will cost $1.8 million. Cue the pricing debate."},{"key":"106_CR21","doi-asserted-by":"publisher","first-page":"495","DOI":"10.3233\/JAD-180370","volume":"67","author":"J Gallacher","year":"2019","unstructured":"Gallacher, J., F. de Reydet de Vulpillieres, B. Amzal, et al. 2019. Challenges for optimizing real-world evidence in Alzheimer's disease: The ROADMAP project. Journal of Alzheimer's Disease 67: 495\u2013501. https:\/\/doi.org\/10.3233\/JAD-180370.","journal-title":"Journal of Alzheimer's Disease"},{"key":"106_CR22","series-title":"STAT","volume-title":"Experimental gene therapy for hemophilia, developed by Pfizer and Sangamo, shows early promise","author":"D Garde","year":"2019","unstructured":"Garde D. Experimental gene therapy for hemophilia, developed by Pfizer and Sangamo, shows early promise. STAT. 2019."},{"key":"106_CR23","doi-asserted-by":"publisher","first-page":"4","DOI":"10.1186\/s13195-017-0332-0","volume":"10","author":"N Geifman","year":"2018","unstructured":"Geifman, N., R.E. Kennedy, L.S. Schneider, et al. 2018. Data-driven identification of endophenotypes of Alzheimer's disease progression: Implications for clinical trials and therapeutic interventions. Alzheimer's Research & Therapy 10: 4. https:\/\/doi.org\/10.1186\/s13195-017-0332-0.","journal-title":"Alzheimer's Research & Therapy"},{"key":"106_CR24","doi-asserted-by":"publisher","first-page":"221","DOI":"10.2147\/CIA.S155145","volume":"13","author":"VV Giau","year":"2018","unstructured":"Giau, V.V., H. Lee, K.H. Shim, et al. 2018. Genome-editing applications of CRISPR-Cas9 to promote in vitro studies of Alzheimer's disease. Clinical Interventions in Aging 13: 221\u2013233. https:\/\/doi.org\/10.2147\/CIA.S155145.","journal-title":"Clinical Interventions in Aging"},{"key":"106_CR25","doi-asserted-by":"publisher","first-page":"31","DOI":"10.1016\/j.yfrne.2018.06.001","volume":"50","author":"H Hampel","year":"2018","unstructured":"Hampel, H., A. Vergallo, F.S. Giorgi, et al. 2018. Precision medicine and drug development in Alzheimer's disease: The importance of sexual dimorphism and patient stratification. Frontiers in Neuroendocrinology 50: 31\u201351. https:\/\/doi.org\/10.1016\/j.yfrne.2018.06.001.","journal-title":"Frontiers in Neuroendocrinology"},{"key":"106_CR26","doi-asserted-by":"publisher","first-page":"143","DOI":"10.1016\/j.neuro.2017.03.006","volume":"61","author":"M Hersi","year":"2017","unstructured":"Hersi, M., B. Irvine, P. Gupta, et al. 2017. Risk factors associated with the onset and progression of Alzheimer's disease: A systematic review of the evidence. Neurotoxicology. 61: 143\u2013187. https:\/\/doi.org\/10.1016\/j.neuro.2017.03.006.","journal-title":"Neurotoxicology."},{"key":"106_CR27","doi-asserted-by":"publisher","first-page":"640","DOI":"10.1377\/hlthaff.2016.1107","volume":"36","author":"J Hsu","year":"2017","unstructured":"Hsu, J., C. Vogeli, M. Price, et al. 2017. Substantial physician turnover and beneficiary \u2018churn\u2019in a large Medicare Pioneer ACO. Health Affairs. 36: 640\u2013648.","journal-title":"Health Affairs."},{"key":"106_CR28","doi-asserted-by":"publisher","first-page":"841","DOI":"10.1111\/jgs.15830","volume":"67","author":"YM Huang","year":"2019","unstructured":"Huang, Y.M., J. Shen, and H.L. Zhao. 2019. Major clinical trials failed the amyloid hypothesis of Alzheimer's disease. Journal of the American Geriatrics Society 67: 841\u2013844. https:\/\/doi.org\/10.1111\/jgs.15830.","journal-title":"Journal of the American Geriatrics Society"},{"key":"106_CR29","volume-title":"Final Evidence Report. (ICER: Institute for Clinical and Economic Review)","author":"ICER","year":"2019","unstructured":"ICER. 2019. Spinraza and Zolgensma for Spinal Muscular Atrophy: Effectiveness and Value. In Final Evidence Report. (ICER: Institute for Clinical and Economic Review)."},{"key":"106_CR30","doi-asserted-by":"publisher","unstructured":"Iqubal, A., M.K. Iqubal, A. Khan, et al. 2020. Gene therapy, a novel therapeutic tool for neurological disorders: Current progress, challenges and future prospective. Current Gene Therapy. https:\/\/doi.org\/10.2174\/1566523220999200716111502.","DOI":"10.2174\/1566523220999200716111502"},{"key":"106_CR31","volume-title":"On Alzheimer\u2019s, scientists head back to the drawing board \u2014 and once-shunned ideas get an audience","author":"M Keshavan","year":"2019","unstructured":"Keshavan M. On Alzheimer\u2019s, scientists head back to the drawing board \u2014 and once-shunned ideas get an audience. STAT. 2019."},{"key":"106_CR32","doi-asserted-by":"publisher","first-page":"729","DOI":"10.3233\/JAD-142262","volume":"44","author":"TK Khan","year":"2015","unstructured":"Khan, T.K., and D.L. Alkon. 2015. Peripheral biomarkers of Alzheimer's disease. Journal of Alzheimer's Disease 44: 729\u2013744. https:\/\/doi.org\/10.3233\/JAD-142262.","journal-title":"Journal of Alzheimer's Disease"},{"key":"106_CR33","doi-asserted-by":"publisher","unstructured":"Ko, Y., and S.M. Chye. 2020. Lifestyle intervention to prevent Alzheimer's disease. Reviews in the Neurosciences. https:\/\/doi.org\/10.1515\/revneuro-2020-0072.","DOI":"10.1515\/revneuro-2020-0072"},{"key":"106_CR34","doi-asserted-by":"publisher","first-page":"373","DOI":"10.3233\/JAD-170705","volume":"61","author":"C Lange","year":"2018","unstructured":"Lange, C., P. Suppa, U. Pietrzyk, et al. 2018. Prediction of Alzheimer's dementia in patients with amnestic mild cognitive impairment in clinical routine: Incremental value of biomarkers of neurodegeneration and brain amyloidosis added stepwise to cognitive status. Journal of Alzheimer's Disease 61: 373\u2013388. https:\/\/doi.org\/10.3233\/JAD-170705.","journal-title":"Journal of Alzheimer's Disease"},{"key":"106_CR35","unstructured":"Loftus, P. 2014. Senate committee is investigating pricing of hepatitis C drug. The Wall Street Journal. 11. https:\/\/www.wsj.com\/articles\/senate-finance-committee-is-investigating-pricing-of-hepatitis-c-drug-1405109206. Accessed 11 Sept 2020."},{"key":"106_CR36","doi-asserted-by":"publisher","DOI":"10.1002\/14651858.CD009628.pub2","volume":"3","author":"G Lombardi","year":"2020","unstructured":"Lombardi, G., G. Crescioli, E. Cavedo, et al. 2020. Structural magnetic resonance imaging for the early diagnosis of dementia due to Alzheimer's disease in people with mild cognitive impairment. Cochrane Database of Systematic Reviews 3: CD009628. https:\/\/doi.org\/10.1002\/14651858.CD009628.pub2.","journal-title":"Cochrane Database of Systematic Reviews"},{"key":"106_CR37","unstructured":"Marchione, M. 2020. Scientists get closer to blood test for Alzheimer\u2019s disease. New York: Associated Press. https:\/\/apnews.com\/4d9f28d2051889e8326f1e9ca5ec5676. Accessed 11 Sept 2020."},{"key":"106_CR38","volume-title":"Novartis wins conditional EU approval for gene therapy Zolgensma","author":"J Miller","year":"2020","unstructured":"Miller J. Novartis wins conditional EU approval for gene therapy Zolgensma. Reuters. 2020."},{"key":"106_CR39","unstructured":"Packer-Tursman J. 2019. Louisiana\u2019s Hepatitis C 'Netflix' Model Sees Positive Results. Washington: AIS Health. https:\/\/aishealth.com\/drug-benefits\/louisianas-hepatitis-c-netflix-model-sees-positive-results\/. Accessed 11 Sept 2020."},{"key":"106_CR40","unstructured":"Pipes S. 2019. Louisiana\u2019s Tries Hard, But Federal Obstacles Cause Hepatitis C Plan to Fall Short. New Jersey: Forbes. https:\/\/www.forbes.com\/sites\/sallypipes\/2019\/08\/07\/louisiana-tries-hard-but-federal-obstacles-cause-hepatitis-c-plan-to-fall-short\/#672603b36ec7. Accessed 11 Sept 2020."},{"key":"106_CR41","doi-asserted-by":"publisher","first-page":"627","DOI":"10.1007\/s12603-018-1023-7","volume":"22","author":"KH Pitk\u00e4l\u00e4","year":"2018","unstructured":"Pitk\u00e4l\u00e4, K.H., F.C. Martin, S. Maggi, et al. 2018 (journal article. Status of Geriatrics in 22 Countries. The Journal of Nutrition, Health & Aging 22: 627\u2013631. https:\/\/doi.org\/10.1007\/s12603-018-1023-7.","journal-title":"The Journal of Nutrition, Health & Aging"},{"key":"106_CR42","doi-asserted-by":"publisher","unstructured":"Prendecki, M., M. Kowalska, E. Toton, et al. 2020. Genetic editing and pharmacogenetics in current and future therapy of neurocognitive disorders. Current Alzheimer Research. https:\/\/doi.org\/10.2174\/1567205017666200422152440.","DOI":"10.2174\/1567205017666200422152440"},{"key":"106_CR43","doi-asserted-by":"publisher","unstructured":"Rabin, J.S., H. Klein, D.R. Kirn, et al. 2019. Associations of physical activity and \u03b2-amyloid with longitudinal cognition and neurodegeneration in clinically normal older AdultsAssociations of physical activity and \u03b2-amyloid with cognition and Neurodegeneration in clinically normal older AdultsAssociations of physical activity and \u03b2-amyloid with cognition and neurodegeneration in clinically normal older adults. JAMA Neurology. https:\/\/doi.org\/10.1001\/jamaneurol.2019.1879.","DOI":"10.1001\/jamaneurol.2019.1879"},{"key":"106_CR44","doi-asserted-by":"publisher","first-page":"321","DOI":"10.3233\/JAD-180422","volume":"65","author":"SP Raikwar","year":"2018","unstructured":"Raikwar, S.P., R. Thangavel, I. Dubova, et al. 2018. Neuro-immuno-gene- and genome-editing-therapy for Alzheimer's disease: Are we there yet? Journal of Alzheimer's Disease 65: 321\u2013344. https:\/\/doi.org\/10.3233\/JAD-180422.","journal-title":"Journal of Alzheimer's Disease"},{"key":"106_CR45","doi-asserted-by":"publisher","first-page":"926","DOI":"10.2174\/1570159X15666170116143743","volume":"15","author":"R Ricciarelli","year":"2017","unstructured":"Ricciarelli, R., and E. Fedele. 2017. The amyloid cascade hypothesis in Alzheimer's disease: It's time to change our mind. Current Neuropharmacology 15: 926\u2013935. https:\/\/doi.org\/10.2174\/1570159X15666170116143743.","journal-title":"Current Neuropharmacology"},{"key":"106_CR46","doi-asserted-by":"publisher","first-page":"390","DOI":"10.3389\/fnagi.2018.00390","volume":"10","author":"BC Riedel","year":"2018","unstructured":"Riedel, B.C., M. Daianu, G. Ver Steeg, et al. 2018. Uncovering biologically coherent peripheral signatures of health and risk for Alzheimer's disease in the aging brain. Frontiers in Aging Neuroscience 10: 390. https:\/\/doi.org\/10.3389\/fnagi.2018.00390.","journal-title":"Frontiers in Aging Neuroscience"},{"key":"106_CR47","doi-asserted-by":"publisher","first-page":"85","DOI":"10.1186\/s13195-017-0312-4","volume":"9","author":"CW Ritchie","year":"2017","unstructured":"Ritchie, C.W., T.C. Russ, S. Banerjee, et al. 2017. The Edinburgh consensus: Preparing for the advent of disease-modifying therapies for Alzheimer's disease. Alzheimer's Research & Therapy 9: 85. https:\/\/doi.org\/10.1186\/s13195-017-0312-4.","journal-title":"Alzheimer's Research & Therapy"},{"key":"106_CR48","doi-asserted-by":"publisher","unstructured":"Salzman, R., F. Cook, T. Hunt, et al. 2018. Addressing the Value of Gene Therapy and Enhancing Patient Access to Transformative Treatments. Molecular Therapy. 26(12):2717\u20132726. https:\/\/doi.org\/10.1016\/j.ymthe.2018.10.017. Epub 2018 Oct 30.","DOI":"10.1016\/j.ymthe.2018.10.017"},{"key":"106_CR49","doi-asserted-by":"publisher","first-page":"207","DOI":"10.2174\/1567205011310020011","volume":"10","author":"A Skoldunger","year":"2013","unstructured":"Skoldunger, A., K. Johnell, B. Winblad, et al. 2013. Mortality and treatment costs have a great impact on the cost-effectiveness of disease modifying treatment in Alzheimer's disease-a simulation study. Current Alzheimer Research. 10: 207\u2013216.","journal-title":"Current Alzheimer Research."},{"key":"106_CR50","doi-asserted-by":"publisher","first-page":"1479","DOI":"10.1056\/NEJMoa1705342","volume":"378","author":"AA Thompson","year":"2018","unstructured":"Thompson, A.A., M.C. Walters, J. Kwiatkowski, et al. 2018. Gene therapy in patients with transfusion-dependent beta-thalassemia. The New England Journal of Medicine 378: 1479\u20131493. https:\/\/doi.org\/10.1056\/NEJMoa1705342.","journal-title":"The New England Journal of Medicine"},{"key":"106_CR51","volume-title":"Celgene exec jumps to head bluebird bio ops in Europe, where its $1.8M gene therapy Zynteglo is now available","author":"A Tong","year":"2020","unstructured":"Tong A. Celgene exec jumps to head bluebird bio ops in Europe, where its $1.8M gene therapy Zynteglo is now available. Endpoints News. 2020."},{"key":"106_CR52","doi-asserted-by":"publisher","first-page":"1977","DOI":"10.1001\/jama.2018.15782","volume":"320","author":"MR Trusheim","year":"2018","unstructured":"Trusheim, M.R., W.M. Cassidy, and P.B. Bach. 2018. Alternative state-level financing for hepatitis C treatment-the \"Netflix model\". JAMA. 320: 1977\u20131978. https:\/\/doi.org\/10.1001\/jama.2018.15782.","journal-title":"JAMA."},{"key":"106_CR53","doi-asserted-by":"publisher","unstructured":"Turner, R.S., M.L. Hebron, A. Lawler, et al. 2020. Nilotinib effects on safety, tolerability, and biomarkers in Alzheimer's disease. Annals of Neurology. https:\/\/doi.org\/10.1002\/ana.25775.","DOI":"10.1002\/ana.25775"},{"key":"106_CR54","doi-asserted-by":"publisher","first-page":"434","DOI":"10.1016\/S1474-4422(18)30053-X","volume":"17","author":"SJ van der Lee","year":"2018","unstructured":"van der Lee, S.J., F.J. Wolters, M.K. Ikram, et al. 2018. The effect of APOE and other common genetic variants on the onset of Alzheimer's disease and dementia: A community-based cohort study. Lancet Neurology 17: 434\u2013444. https:\/\/doi.org\/10.1016\/S1474-4422(18)30053-X.","journal-title":"Lancet Neurology"},{"key":"106_CR55","unstructured":"Voretigene neparvovec-rzyl (Luxturna) for inherited retinal dystrophy. 2018. The Medical Letter on Drugs and Therapeutics 60:53\u201355. https:\/\/pubmed.ncbi.nlm.nih.gov\/29635265\/"},{"key":"106_CR56","doi-asserted-by":"publisher","first-page":"1311","DOI":"10.3233\/jad-180865","volume":"68","author":"AD Watt","year":"2019","unstructured":"Watt, A.D., N.L. Jenkins, G. McColl, et al. 2019. Ethical issues in the treatment of late-stage Alzheimer's disease. Journal of Alzheimer's Disease 68: 1311\u20131316. https:\/\/doi.org\/10.3233\/jad-180865.","journal-title":"Journal of Alzheimer's Disease"},{"key":"106_CR57","doi-asserted-by":"publisher","first-page":"S41","DOI":"10.3233\/JAD-179905","volume":"64","author":"A Wimo","year":"2018","unstructured":"Wimo, A. 2018. The end of the beginning of the Alzheimer's disease nightmare: A Devil's Advocate's view. Journal of Alzheimer's Disease 64: S41\u2013S46. https:\/\/doi.org\/10.3233\/JAD-179905.","journal-title":"Journal of Alzheimer's Disease"}],"container-title":["Life Sciences, Society and Policy"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1186\/s40504-020-00106-2.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/article\/10.1186\/s40504-020-00106-2\/fulltext.html","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1186\/s40504-020-00106-2.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2021,10,12]],"date-time":"2021-10-12T05:34:34Z","timestamp":1634016874000},"score":1,"resource":{"primary":{"URL":"https:\/\/lsspjournal.biomedcentral.com\/articles\/10.1186\/s40504-020-00106-2"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2020,10,12]]},"references-count":57,"journal-issue":{"issue":"1","published-print":{"date-parts":[[2020,12]]}},"alternative-id":["106"],"URL":"https:\/\/doi.org\/10.1186\/s40504-020-00106-2","relation":{},"ISSN":["2195-7819"],"issn-type":[{"value":"2195-7819","type":"electronic"}],"subject":[],"published":{"date-parts":[[2020,10,12]]},"assertion":[{"value":"19 June 2020","order":1,"name":"received","label":"Received","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"9 September 2020","order":2,"name":"accepted","label":"Accepted","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"12 October 2020","order":3,"name":"first_online","label":"First Online","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"MA is a part-time paid consultant to Variant Bio and Genetic Alliance.OC-F and BK are part-time paid consultants to Seelos Therapeutics.","order":1,"name":"Ethics","group":{"name":"EthicsHeading","label":"Competing interests"}}],"article-number":"11"}}