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Comparative Effecti...
Comparative Effectiveness of Autologous Hematopoietic Stem Cell Transplant vs Fingolimod, Natalizumab, and Ocrelizumab in Highly Active Relapsing-Remitting Multiple Sclerosis
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- Kalincik, Tomas (författare)
- Royal Melbourne Hosp, Dept Neurol, Ctr Neuroimmunol, Melbourne, Vic, Australia.;Univ Melbourne, Dept Med, Melbourne, Vic, Australia.
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- Sharmin, Sifat (författare)
- Royal Melbourne Hosp, Dept Neurol, Ctr Neuroimmunol, Melbourne, Vic, Australia.;Univ Melbourne, Dept Med, Melbourne, Vic, Australia.
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- Roos, Izanne (författare)
- Royal Melbourne Hosp, Dept Neurol, Ctr Neuroimmunol, Melbourne, Vic, Australia.;Univ Melbourne, Dept Med, Melbourne, Vic, Australia.
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visa fler...
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- Freedman, Mark S. (författare)
- Univ Ottawa, Ottawa Hosp Res Inst, Dept Med, Ottawa, ON, Canada.
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- Atkins, Harold (författare)
- Univ Ottawa, Ottawa Hosp Res Inst, Ottawa, ON, Canada.
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- Burman, Joachim, 1974- (författare)
- Uppsala universitet,Experimentell neurologi
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- Massey, Jennifer (författare)
- St Vincents Hosp Sydney, Dept Neurol, Sydney, NSW, Australia.;Univ New South Wales, St Vincents Clin Sch, Sydney, NSW, Australia.
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- Sutton, Ian (författare)
- St Vincents Hosp Sydney, Dept Neurol, Sydney, NSW, Australia.;Univ Sydney, Sydney, NSW, Australia.
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- Withers, Barbara (författare)
- Univ New South Wales, St Vincents Clin Sch, Sydney, NSW, Australia.;St Vincents Hosp Sydney, Dept Haematol, Sydney, NSW, Australia.
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- Macdonell, Richard (författare)
- Austin Hlth, Dept Neurol, Melbourne, Vic, Australia.;Univ Melbourne, Melbourne, Vic, Australia.
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- Grigg, Andrew (författare)
- Univ Melbourne, Melbourne, Vic, Australia.;Austin Hlth, Dept Haematol, Melbourne, Vic, Australia.
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- Torkildsen, Øivind (författare)
- Haukeland Hosp, Dept Neurol, Bergen, Norway.
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- Bo, Lars (författare)
- Haukeland Hosp, Dept Neurol, Bergen, Norway.
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- Lehmann, Anne Kristine (författare)
- Haukeland Hosp, Dept Haematol, Bergen, Norway.
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- Havrdova, Eva Kubala (författare)
- Charles Univ Prague, Dept Neurol, Fac Med 1, Prague, Czech Republic.;Gen Univ Hosp, Prague, Czech Republic.
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- Krasulova, Eva (författare)
- Charles Univ Prague, Dept Neurol, Fac Med 1, Prague, Czech Republic.;Gen Univ Hosp, Prague, Czech Republic.
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- Trneny, Marek (författare)
- Gen Univ Hosp, Prague, Czech Republic.;Charles Univ Prague, Fac Med 1, Dept Haematol, Prague, Czech Republic.
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- Kozak, Tomas (författare)
- Charles Univ Prague, Dept Haematol, Fac Med 3, Prague, Czech Republic.;Univ Hosp Kralovske Vinohrady, Prague, Czech Republic.
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- van der Walt, Anneke (författare)
- Alfred Hosp, Dept Neurol, Melbourne, Vic, Australia.;Monash Univ, Cent Clin Sch, Melbourne, Vic, Australia.
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- Butzkueven, Helmut (författare)
- Alfred Hosp, Dept Neurol, Melbourne, Vic, Australia.;Monash Univ, Cent Clin Sch, Melbourne, Vic, Australia.
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- McCombe, Pamela (författare)
- Univ Queensland, Brisbane, Qld, Australia.;Royal Brisbane & Womens Hosp, Brisbane, Qld, Australia.
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- Skibina, Olga (författare)
- Alfred Hosp, Dept Neurol, Melbourne, Vic, Australia.;Box Hill Hosp, Dept Neurol, Melbourne, Vic, Australia.;Monash Univ, Melbourne, Vic, Australia.
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- Lechner-Scott, Jeannette (författare)
- Univ Newcastle, Sch Med & Publ Hlth, Newcastle, NSW, Australia.;Hunter New England Hlth, Dept Neurol, John Hunter Hosp, Newcastle, NSW, Australia.
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- Willekens, Barbara (författare)
- Antwerp Univ Hosp, Dept Neurol, Edegem, Belgium.;Univ Antwerp, Translat Neurosci Res Grp, Fac Med & Hlth Sci, Antwerp, Belgium.
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- Cartechini, Elisabetta (författare)
- Azienda Sanit Unica Reg Marche AV3, UOC Neurol, Macerata, Italy.
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- Ozakbas, Serkan (författare)
- Dokuz Eylul Univ, Izmir, Turkiye.
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- Alroughani, Raed (författare)
- Amiri Hosp, Div Neurol, Dept Med, Sharq, Kuwait.
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- Kuhle, Jens (författare)
- Univ Hosp, Neurol Clin & Policlin, Dept Med, Basel, Switzerland.;Univ Hosp, Neurol Clin & Policlin, Dept Clin Res, Basel, Switzerland.;Univ Basel, Basel, Switzerland.
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- Patti, Francesco (författare)
- GF Ingrassia, Dept Med & Surg Sci & Adv Technol, Catania, Italy.;Univ Catania, Multiple Sclerosis Ctr, Catania, Italy.
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- Duquette, Pierre (författare)
- CHUM MS Ctr, Montreal, PQ, Canada.;Univ Montreal, Montreal, PQ, Canada.
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- Lugaresi, Alessandra (författare)
- IRCCS Ist Sci Neurol Bologna, Bologna, Italy.;Univ Bologna, Dipartimento Sci Biomed & Neuromotorie, Bologna, Italy.
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- Khoury, Samia J. (författare)
- Amer Univ Beirut, Med Ctr, Nehme & Therese Tohme Multiple Sclerosis Ctr, Beirut, Lebanon.
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- Slee, Mark (författare)
- Flinders Univ S Australia, Adelaide, SA, Australia.
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- Turkoglu, Recai (författare)
- Haydarpasa Numune Training & Res Hosp, Istanbul, Turkiye.
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- Hodgkinson, Suzanne (författare)
- Liverpool Hosp, Sydney, NSW, Australia.
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- John, Nevin (författare)
- Monash Med Ctr, Melbourne, Vic, Australia.;Monash Univ, Dept Med, Sch Clin Sci, Melbourne, Vic, Australia.
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- Maimone, Davide (författare)
- Garibaldi Hosp, Catania, Italy.
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- Sa, Maria Jose (författare)
- Ctr Hosp Univ Sao Joao, Dept Neurol, Porto, Portugal.
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- van Pesch, Vincent (författare)
- Clin Univ St Luc, Brussels, Belgium.;Catholic Univ Louvain, Ottignies, Belgium.
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- Gerlach, Oliver (författare)
- Zuyderland Med Ctr, Acad MS Ctr Zuyderland, Dept Neurol, Sittard Geleen, Belgium.;Maastricht Univ, Sch Mental Hlth & Neurosci, Maastricht, Netherlands.
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- Laureys, Guy (författare)
- Univ Hosp Ghent, Dept Neurol, Ghent, Belgium.
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- Van Hijfte, Liesbeth (författare)
- Univ Hosp Ghent, Dept Neurol, Ghent, Belgium.
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- Karabudak, Rana (författare)
- Hacettepe Univ Hosp, Dept Neurol, Ankara, Turkiye.
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- Spitaleri, Daniele (författare)
- Azienda Osped Rilievo Nazl San Giuseppe Moscati A, Avellino, Italy.
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- Csepany, Tunde (författare)
- Univ Debrecen, Dept Neurol, Fac Med, Debrecen, Hungary.
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- Gouider, Riadh (författare)
- Razi Univ Hosp, Dept Neurol, Tunis, Tunisia.;Univ Tunis El Manar, Fac Med Tunis, Tunis, Tunisia.
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- Castillo-Trivino, Tamara (författare)
- Hosp Univ Donostia, San Sebastian, Spain.
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- Taylor, Bruce (författare)
- Univ Tasmania, Menzies Inst Med Res, Hobart, Tas, Australia.;Royal Hobart Hosp, Hobart, Tas, Australia.
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- Sharrack, Basil (författare)
- Sheffield Teaching Hosp NHS Fdn Trust, Dept Neurol, Sheffield, S Yorkshire, England.
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- Snowden, John A. (författare)
- Sheffield Teaching Hosp NHS Fdn Trust, Dept Haematol, Sheffield, S Yorkshire, England.
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Royal Melbourne Hosp, Dept Neurol, Ctr Neuroimmunol, Melbourne, Vic, Australia;Univ Melbourne, Dept Med, Melbourne, Vic, Australia. Univ Ottawa, Ottawa Hosp Res Inst, Dept Med, Ottawa, ON, Canada. (creator_code:org_t)
- American Medical Association (AMA), 2023
- 2023
- Engelska.
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Ingår i: JAMA Neurology. - : American Medical Association (AMA). - 2168-6149 .- 2168-6157. ; 80:7, s. 702-713
- Relaterad länk:
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https://urn.kb.se/re...
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https://doi.org/10.1...
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Abstract
Ämnesord
Stäng
- Importance Autologous hematopoietic stem cell transplant (AHSCT) is available for treatment of highly active multiple sclerosis (MS).Objective To compare the effectiveness of AHSCT vs fingolimod, natalizumab, and ocrelizumab in relapsing-remitting MS by emulating pairwise trials.Design, Setting, and Participants This comparative treatment effectiveness study included 6 specialist MS centers with AHSCT programs and international MSBase registry between 2006 and 2021. The study included patients with relapsing-remitting MS treated with AHSCT, fingolimod, natalizumab, or ocrelizumab with 2 or more years study follow-up including 2 or more disability assessments. Patients were matched on a propensity score derived from clinical and demographic characteristics.Exposure AHSCT vs fingolimod, natalizumab, or ocrelizumab.Main outcomes Pairwise-censored groups were compared on annualized relapse rates (ARR) and freedom from relapses and 6-month confirmed Expanded Disability Status Scale (EDSS) score worsening and improvement.Results Of 4915 individuals, 167 were treated with AHSCT; 2558, fingolimod; 1490, natalizumab; and 700, ocrelizumab. The prematch AHSCT cohort was younger and with greater disability than the fingolimod, natalizumab, and ocrelizumab cohorts; the matched groups were closely aligned. The proportion of women ranged from 65% to 70%, and the mean (SD) age ranged from 35.3 (9.4) to 37.1 (10.6) years. The mean (SD) disease duration ranged from 7.9 (5.6) to 8.7 (5.4) years, EDSS score ranged from 3.5 (1.6) to 3.9 (1.9), and frequency of relapses ranged from 0.77 (0.94) to 0.86 (0.89) in the preceding year. Compared with the fingolimod group (769 [30.0%]), AHSCT (144 [86.2%]) was associated with fewer relapses (ARR: mean [SD], 0.09 [0.30] vs 0.20 [0.44]), similar risk of disability worsening (hazard ratio [HR], 1.70; 95% CI, 0.91-3.17), and higher chance of disability improvement (HR, 2.70; 95% CI, 1.71-4.26) over 5 years. Compared with natalizumab (730 [49.0%]), AHSCT (146 [87.4%]) was associated with marginally lower ARR (mean [SD], 0.08 [0.31] vs 0.10 [0.34]), similar risk of disability worsening (HR, 1.06; 95% CI, 0.54-2.09), and higher chance of disability improvement (HR, 2.68; 95% CI, 1.72-4.18) over 5 years. AHSCT (110 [65.9%]) and ocrelizumab (343 [49.0%]) were associated with similar ARR (mean [SD], 0.09 [0.34] vs 0.06 [0.32]), disability worsening (HR, 1.77; 95% CI, 0.61-5.08), and disability improvement (HR, 1.37; 95% CI, 0.66-2.82) over 3 years. AHSCT-related mortality occurred in 1 of 159 patients (0.6%).Conclusion In this study, the association of AHSCT with preventing relapses and facilitating recovery from disability was considerably superior to fingolimod and marginally superior to natalizumab. This study did not find evidence for difference in the effectiveness of AHSCT and ocrelizumab over a shorter available follow-up time.
Ämnesord
- MEDICIN OCH HÄLSOVETENSKAP -- Klinisk medicin -- Neurologi (hsv//swe)
- MEDICAL AND HEALTH SCIENCES -- Clinical Medicine -- Neurology (hsv//eng)
- MEDICIN OCH HÄLSOVETENSKAP -- Hälsovetenskap -- Hälso- och sjukvårdsorganisation, hälsopolitik och hälsoekonomi (hsv//swe)
- MEDICAL AND HEALTH SCIENCES -- Health Sciences -- Health Care Service and Management, Health Policy and Services and Health Economy (hsv//eng)
- MEDICIN OCH HÄLSOVETENSKAP -- Medicinska och farmaceutiska grundvetenskaper -- Neurovetenskaper (hsv//swe)
- MEDICAL AND HEALTH SCIENCES -- Basic Medicine -- Neurosciences (hsv//eng)
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- Av författaren/redakt...
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Kalincik, Tomas
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Sharmin, Sifat
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Roos, Izanne
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Freedman, Mark S ...
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Atkins, Harold
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Burman, Joachim, ...
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visa fler...
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Massey, Jennifer
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Sutton, Ian
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Withers, Barbara
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Macdonell, Richa ...
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Grigg, Andrew
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Torkildsen, Øivi ...
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Bo, Lars
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Lehmann, Anne Kr ...
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Havrdova, Eva Ku ...
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Krasulova, Eva
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Trneny, Marek
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Kozak, Tomas
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van der Walt, An ...
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Butzkueven, Helm ...
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McCombe, Pamela
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Skibina, Olga
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Lechner-Scott, J ...
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Willekens, Barba ...
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Cartechini, Elis ...
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Ozakbas, Serkan
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Alroughani, Raed
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Kuhle, Jens
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Patti, Francesco
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Duquette, Pierre
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Lugaresi, Alessa ...
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Khoury, Samia J.
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Slee, Mark
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Turkoglu, Recai
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Hodgkinson, Suza ...
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John, Nevin
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Maimone, Davide
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Sa, Maria Jose
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van Pesch, Vince ...
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Gerlach, Oliver
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Laureys, Guy
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Van Hijfte, Lies ...
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Karabudak, Rana
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Spitaleri, Danie ...
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Csepany, Tunde
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Gouider, Riadh
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Castillo-Trivino ...
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Taylor, Bruce
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Sharrack, Basil
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Snowden, John A.
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- MEDICIN OCH HÄLSOVETENSKAP
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MEDICIN OCH HÄLS ...
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och Klinisk medicin
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och Neurologi
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- MEDICIN OCH HÄLSOVETENSKAP
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MEDICIN OCH HÄLS ...
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och Hälsovetenskap
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och Hälso och sjukvå ...
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- MEDICIN OCH HÄLSOVETENSKAP
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MEDICIN OCH HÄLS ...
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och Medicinska och f ...
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och Neurovetenskaper
- Artiklar i publikationen
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JAMA Neurology
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Uppsala universitet