المؤلفون: Monteleone, Berrin, Forster, Katie, Chua, Gin Nie, Zhang, Rongrong, Lloyd, Andrew, Castellano, Paul, Tomazos, Ioannis

المساهمون: PTC Therapeutics Inc

المصدر: Health and Quality of Life Outcomes ; volume 23, issue 1 ; ISSN 1477-7525

الاتاحة: https://doi.org/10.1186/s12955-025-02335-5
https://link.springer.com/content/pdf/10.1186/s12955-025-02335-5.pdf
https://link.springer.com/article/10.1186/s12955-025-02335-5/fulltext.html

المؤلفون: Landfeldt, Erik, Aleman, A., Abner, Sophie, Zhang, R., Werner, C., Tomazos, Ioannis, Lochmüller, Hanns, Quinlivan, Ros

المصدر: Journal of neuromuscular diseases. - 11, 1 (2024) , 25-57, ISSN: 2214-3602

وصف الملف: pdf

Relation: https://freidok.uni-freiburg.de/data/241556

الاتاحة: https://freidok.uni-freiburg.de/data/241556
https://nbn-resolving.org/urn:nbn:de:bsz:25-freidok-2415565
https://doi.org/10.3233/jnd-230094
https://freidok.uni-freiburg.de/dnb/download/241556

المؤلفون: Landfeldt, Erik, Aleman, Alberto, Abner, Sophia C., Zhang, R., Werner, Christian, Tomazos, Ioannis, Ferizovic, Nermina, Lochmüller, Hanns, Kirschner, Janbernd

المصدر: Journal of neuromuscular diseases. - 11, 3 (2024) , 579-612, ISSN: 2214-3602

وصف الملف: pdf

Relation: https://freidok.uni-freiburg.de/data/248777

الاتاحة: https://freidok.uni-freiburg.de/data/248777
https://nbn-resolving.org/urn:nbn:de:bsz:25-freidok-2487775
https://doi.org/10.3233/jnd-230220
https://freidok.uni-freiburg.de/dnb/download/248777

المؤلفون: Greenbaum, Larry A., Licht, Christoph, Nikolaou, Vasileios, Al-Dakkak, Imad, Green, Janet, Haas, Christian Stefan, Román-Ortiz, Elena, Cheong, Hae Il, Sartz, Lisa, Swinford, Rita, Tomazos, Ioannis, Miller, Benjamin, Cataland, Spero

المصدر: Kidney International Reports. 5(8):1161-1171

مصطلحات موضوعية: aHUS, atypical hemolytic uremic syndrome, complement, FACIT-Fatigue, fatigue, patient-reported outcomes, Medicin och hälsovetenskap, Klinisk medicin, Urologi och njurmedicin, Medical and Health Sciences, Clinical Medicine, Urology and Nephrology

URL الوصول: https://lup.lub.lu.se/record/7ffe7333-9451-492e-9bd8-32d7c187edaa
http://dx.doi.org/10.1016/j.ekir.2020.05.003

المؤلفون: Schrezenmeier, Hubert, Kulasekararaj, Austin, Mitchell, Lindsay, de Latour, Régis Peffault, Devos, Timothy, Okamoto, Shinichiro, Wells, Richard, Popoff, Evan, Cheung, Antoinette, Wang, Alice, Tomazos, Ioannis, Patel, Yogesh, Lee, Jong Wook

المساهمون: Alexion Pharmaceuticals, Universität Ulm

المصدر: Annals of Hematology ; volume 103, issue 1, page 5-15 ; ISSN 0939-5555 1432-0584

الاتاحة: http://dx.doi.org/10.1007/s00277-023-05483-0
https://link.springer.com/content/pdf/10.1007/s00277-023-05483-0.pdf
https://link.springer.com/article/10.1007/s00277-023-05483-0/fulltext.html

المؤلفون: Bertini, Enrico, Gregg, Emily, Bartlett, Chris, Senthilnathan, Vij, Arber, Mick, Watkins, Deborah, Graziadio, Sara, Tomazos, Ioannis

المساهمون: PTC Therapeutics

المصدر: Orphanet Journal of Rare Diseases ; volume 18, issue 1 ; ISSN 1750-1172

الاتاحة: http://dx.doi.org/10.1186/s13023-023-02945-6
https://link.springer.com/content/pdf/10.1186/s13023-023-02945-6.pdf
https://link.springer.com/article/10.1186/s13023-023-02945-6/fulltext.html

المؤلفون: Wang, Yan, Al-Dakkak, Imad, Garlo, Katherine, Ong, Moh-Lim, Tomazos, Ioannis, Mahajerin, Arash

المساهمون: PTC Therapeutics

المصدر: Kidney Medicine ; volume 5, issue 8, page 100683 ; ISSN 2590-0595

الاتاحة: http://dx.doi.org/10.1016/j.xkme.2023.100683
https://api.elsevier.com/content/article/PII:S2590059523000997?httpAccept=text/xml
https://api.elsevier.com/content/article/PII:S2590059523000997?httpAccept=text/plain

المؤلفون: Landfeldt, Erik, Alemán, Alberto, Abner, Sophia, Zhang, Rongrong, Werner, Christian, Tomazos, Ioannis, Lochmüller, Hanns, Quinlivan, Ros M., Wahbi, Karim

المصدر: Orphanet Journal of Rare Diseases; 9/28/2024, Vol. 19 Issue 1, p1-34, 34p

مصطلحات موضوعية: LEFT ventricular dysfunction, DUCHENNE muscular dystrophy, CONSCIOUSNESS raising, MINERALOCORTICOID receptors, VENTRICULAR dysfunction, ALDOSTERONE antagonists

المؤلفون: Shammo,Jamile, Gajra,Ajeet, Patel,Yogesh, Tomazos,Ioannis, Kish,Jonathan, Hill,Anita, Sierra,J Rafael, Araten,David

مصطلحات موضوعية: Journal of Blood Medicine

وصف الملف: text/html

Relation: https://www.dovepress.com/low-rate-of-clinically-evident-extravascular-hemolysis-in-patients-wit-peer-reviewed-fulltext-article-JBM

الاتاحة: https://www.dovepress.com/low-rate-of-clinically-evident-extravascular-hemolysis-in-patients-wit-peer-reviewed-fulltext-article-JBM

المؤلفون: Shammo, Jamile, Gajra, Ajeet, Patel, Yogesh, Tomazos, Ioannis, Kish, Jonathan, Hill, Anita, Sierra, J Rafael, Araten, David

المصدر: Journal of Blood Medicine ; volume Volume 13, page 425-437 ; ISSN 1179-2736

الاتاحة: http://dx.doi.org/10.2147/jbm.s361863
https://www.dovepress.com/getfile.php?fileID=82972

المؤلفون: Levy, Adrian R., Chen, Peter, Johnston, Karissa, Wang, Yan, Popoff, Evan, Tomazos, Ioannis

المساهمون: Alexion, AstraZeneca Rare Disease

المصدر: Journal of Medical Economics ; volume 25, issue 1, page 249-259 ; ISSN 1369-6998 1941-837X

الاتاحة: http://dx.doi.org/10.1080/13696998.2022.2027706
https://www.tandfonline.com/doi/pdf/10.1080/13696998.2022.2027706

المؤلفون: Doll, Helen, Coşkun, Ufuk, Hartford, Chris, Tomazos, Ioannis

المساهمون: Alexion Pharmaceuticals

المصدر: Journal of Patient-Reported Outcomes ; volume 5, issue 1 ; ISSN 2509-8020

الاتاحة: http://dx.doi.org/10.1186/s41687-021-00319-9
https://link.springer.com/content/pdf/10.1186/s41687-021-00319-9.pdf
https://link.springer.com/article/10.1186/s41687-021-00319-9/fulltext.html

المؤلفون: Kaiser,Karen, Yount,Susan E, Martens,Christa E, Webster,Kimberly A, Shaunfield,Sara, Sparling,Amy, Peipert,John Devin, Cella,David, Rottinghaus,Scott T, Donato,Bonnie MK, Wells,Richard, Tomazos,Ioannis

مصطلحات موضوعية: Patient Preference and Adherence

وصف الملف: text/html

Relation: https://www.dovepress.com/assessing-preferences-for-rare-disease-treatment-qualitative-developme-peer-reviewed-fulltext-article-PPA

الاتاحة: https://www.dovepress.com/assessing-preferences-for-rare-disease-treatment-qualitative-developme-peer-reviewed-fulltext-article-PPA

المؤلفون: O’Connell, Thomas, Buessing, Marric, Johnson, Scott, Tu, Lufei, Thomas, Simu K., Tomazos, Ioannis

المساهمون: Alexion Pharmaceuticals

المصدر: PharmacoEconomics ; volume 38, issue 9, page 981-994 ; ISSN 1170-7690 1179-2027

الاتاحة: http://dx.doi.org/10.1007/s40273-020-00929-z
https://link.springer.com/content/pdf/10.1007/s40273-020-00929-z.pdf
https://link.springer.com/article/10.1007/s40273-020-00929-z/fulltext.html

المؤلفون: Peipert, John Devin, Kulasekararaj, Austin G., Gaya, Anna, Langemeijer, Saskia M. C., Yount, Susan, Gonzalez-Fernandez, F. Ataulfo, Ojeda Gutierrez, Emilio, Martens, Christa, Sparling, Amy, Webster, Kimberly A., Cella, David, Tomazos, Ioannis, Ogawa, Masayo, Piatek, Caroline I., Wells, Richard, Sicre de Fontbrune, Flore, Röth, Alexander, Mitchell, Lindsay, Hill, Anita, Kaiser, Karen

المساهمون: Dingli, David, Alexion Pharmaceuticals

المصدر: PLOS ONE ; volume 15, issue 9, page e0237497 ; ISSN 1932-6203

الاتاحة: http://dx.doi.org/10.1371/journal.pone.0237497
https://dx.plos.org/10.1371/journal.pone.0237497

المؤلفون: Wang, Yan, Johnston, Karissa, Popoff, Evan, Myren, Karl-Johan, Cheung, Antoinette, Faria, Claudio, Tomazos, Ioannis

المصدر: Journal of Medical Economics ; volume 23, issue 12, page 1503-1515 ; ISSN 1369-6998 1941-837X

الاتاحة: http://dx.doi.org/10.1080/13696998.2020.1831519
https://www.tandfonline.com/doi/pdf/10.1080/13696998.2020.1831519

المؤلفون: Kaiser, Karen, Yount, Susan E, Martens, Christa E, Webster, Kimberly A, Shaunfield, Sara, Sparling, Amy, Peipert, John Devin, Cella, David, Rottinghaus, Scott T, Donato, Bonnie MK, Wells, Richard, Tomazos, Ioannis

المصدر: Patient Preference and Adherence ; volume Volume 14, page 705-715 ; ISSN 1177-889X

الاتاحة: http://dx.doi.org/10.2147/ppa.s233830
https://www.dovepress.com/getfile.php?fileID=57260

المؤلفون: Tomazos, Ioannis, Sierra, J. Rafael, Johnston, Karissa M., Cheung, Antoinette, Brodsky, Robert A., Weitz, Ilene C.

المساهمون: Alexion Pharmaceuticals

المصدر: Hematology ; volume 25, issue 1, page 327-334 ; ISSN 1607-8454

الاتاحة: http://dx.doi.org/10.1080/16078454.2020.1807226
https://www.tandfonline.com/doi/pdf/10.1080/16078454.2020.1807226

المؤلفون: Schrezenmeier, Hubert, Kulasekararaj, Austin, Mitchell, Lindsay, de Latour, Régis Peffault, Devos, Timothy, Okamoto, Shinichiro, Wells, Richard, Popoff, Evan, Cheung, Antoinette, Wang, Alice, Tomazos, Ioannis, Patel, Yogesh, Lee, Jong Wook

المصدر: Annals of Hematology; Jan2024, Vol. 103 Issue 1, p5-15, 11p

مصطلحات موضوعية: PAROXYSMAL hemoglobinuria, ECULIZUMAB, PATIENT reported outcome measures, FATIGUE (Physiology), REGRESSION analysis, LACTATE dehydrogenase

المؤلفون: Szabo, Shelagh M., Tomazos, Ioannis C., Petryk, Anna, Powell, Lauren C., Donato, Bonnie M.K., Zarate, Yuri A., Tiulpakov, Anatoly, Martos-Moreno, Gabriel Ángel

المساهمون: UAM. Departamento de Pediatría, Instituto de Investigación del Hospital de La Princesa (IP)

مصطلحات موضوعية: Burden, Hypophosphatasia, Manifestations, Signs, Symptoms, Systematic literature review, Medicina

وصف الملف: application/pdf

Relation: Orphanet Journal of Rare Diseases; https://doi.org/10.1186/s13023-019-1062-0; Orphanet Journal of Rare Diseases 14.1 (2019): 85; http://hdl.handle.net/10486/689105; 85-1; 85-9; 14

الاتاحة: http://hdl.handle.net/10486/689105
https://doi.org/10.1186/s13023-019-1062-0