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Page 1
Long-term safety and outcomes in hereditary tyrosinaemia type 1 with nitisinone treatment: a 15-year non-interventional, multicentre study.
Spiekerkoetter U, Couce ML, Das AM, de Laet C, Dionisi-Vici C, Lund AM, Schiff M, Spada M, Sparve E, Szamosi J, Vara R, Rudebeck M. Spiekerkoetter U, et al. Among authors: rudebeck m. Lancet Diabetes Endocrinol. 2021 Jul;9(7):427-435. doi: 10.1016/S2213-8587(21)00092-9. Epub 2021 May 21. Lancet Diabetes Endocrinol. 2021. PMID: 34023005 Clinical Trial.
Suitability Of Nitisinone In Alkaptonuria 1 (SONIA 1): an international, multicentre, randomised, open-label, no-treatment controlled, parallel-group, dose-response study to investigate the effect of once daily nitisinone on 24-h urinary homogentisic acid excretion in patients with alkaptonuria after 4 weeks of treatment.
Ranganath LR, Milan AM, Hughes AT, Dutton JJ, Fitzgerald R, Briggs MC, Bygott H, Psarelli EE, Cox TF, Gallagher JA, Jarvis JC, van Kan C, Hall AK, Laan D, Olsson B, Szamosi J, Rudebeck M, Kullenberg T, Cronlund A, Svensson L, Junestrand C, Ayoob H, Timmis OG, Sireau N, Le Quan Sang KH, Genovese F, Braconi D, Santucci A, Nemethova M, Zatkova A, McCaffrey J, Christensen P, Ross G, Imrich R, Rovensky J. Ranganath LR, et al. Among authors: rudebeck m. Ann Rheum Dis. 2016 Feb;75(2):362-7. doi: 10.1136/annrheumdis-2014-206033. Epub 2014 Dec 4. Ann Rheum Dis. 2016. PMID: 25475116 Clinical Trial.
Efficacy and safety of once-daily nitisinone for patients with alkaptonuria (SONIA 2): an international, multicentre, open-label, randomised controlled trial.
Ranganath LR, Psarelli EE, Arnoux JB, Braconi D, Briggs M, Bröijersén A, Loftus N, Bygott H, Cox TF, Davison AS, Dillon JP, Fisher M, FitzGerald R, Genovese F, Glasova H, Hall AK, Hughes AT, Hughes JH, Imrich R, Jarvis JC, Khedr M, Laan D, Le Quan Sang KH, Luangrath E, Lukáčová O, Milan AM, Mistry A, Mlynáriková V, Norman BP, Olsson B, Rhodes NP, Rovenský J, Rudebeck M, Santucci A, Shweihdi E, Scott C, Sedláková J, Sireau N, Stančík R, Szamosi J, Taylor S, van Kan C, Vinjamuri S, Vrtíková E, Webb C, West E, Záňová E, Zatkova A, Gallagher JA. Ranganath LR, et al. Among authors: rudebeck m. Lancet Diabetes Endocrinol. 2020 Sep;8(9):762-772. doi: 10.1016/S2213-8587(20)30228-X. Epub 2020 Aug 18. Lancet Diabetes Endocrinol. 2020. PMID: 32822600 Clinical Trial.
Determinants of tyrosinaemia during nitisinone therapy in alkaptonuria.
Ranganath LR, Milan AM, Hughes AT, Davison AS, M K, Norman BP, Bou-Gharios G, Gallagher JA, Imrich R, Arnoux JB, Rudebeck M, Olsson B. Ranganath LR, et al. Among authors: rudebeck m. Sci Rep. 2022 Sep 27;12(1):16083. doi: 10.1038/s41598-022-20424-z. Sci Rep. 2022. PMID: 36167967 Free PMC article.
Safety, Pharmacokinetics, and Efficacy of Palifermin in Children and Adolescents with Acute Leukemias Undergoing Myeloablative Therapy and Allogeneic Hematopoietic Stem Cell Transplantation: A Pediatric Blood and Marrow Transplant Consortium Trial.
Morris J, Rudebeck M, Neudorf S, Moore T, Duerst R, Shah AJ, Graham M, Aquino V, Morris C, Olsson B. Morris J, et al. Among authors: rudebeck m. Biol Blood Marrow Transplant. 2016 Jul;22(7):1247-1256. doi: 10.1016/j.bbmt.2016.02.016. Epub 2016 Mar 8. Biol Blood Marrow Transplant. 2016. PMID: 26968792 Free article. Clinical Trial.
A Prospective Cohort Study Comparing Long-Term Outcomes with and without Palifermin in Patients Receiving Hematopoietic Cell Transplantation for Hematologic Malignancies.
Saber W, Steinert P, Zhang MJ, Chen M, Pope A, Keating A, Wingard JR, Ballen K, Stiff P, Perales MA, Forman S, Champlin R, Langston A, Rudebeck M, Horowitz M. Saber W, et al. Among authors: rudebeck m. Transplant Cell Ther. 2021 Oct;27(10):837.e1-837.e10. doi: 10.1016/j.jtct.2021.06.028. Epub 2021 Jul 2. Transplant Cell Ther. 2021. PMID: 34224914 Free PMC article.
Characterization of changes in the tyrosine pathway by 24-h profiling during nitisinone treatment in alkaptonuria.
Ranganath LR, Milan AM, Hughes AT, Davison AS, Khedr M, Norman BP, Bou-Gharios G, Gallagher JA, Gornall M, Jackson R, Imrich R, Rovensky J, Rudebeck M, Olsson B. Ranganath LR, et al. Among authors: rudebeck m. Mol Genet Metab Rep. 2022 Feb 1;30:100846. doi: 10.1016/j.ymgmr.2022.100846. eCollection 2022 Mar. Mol Genet Metab Rep. 2022. PMID: 35242577 Free PMC article.
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