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, Lakshminarayan R, Milan, Anna M, Hughes, Andrew T, Dutton, John J, Fitzgerald, Richard, Briggs, Michael C, Bygott, Helen, Psarelli, Eftychia E, Cox, Trevor F, Gallagher, James A, Jarvis, Jonathan C, van Kan, Christa, Hall, Anthony K, Laan, Dinny, Olsson, Birgitta, Szamosi, Johan, Rudebeck, Mattias, Kullenberg, Torbjörn, Cronlund, Arvid, Svensson, Lennart, Junestrand, Carin, Ayoob, Hana, Timmis, Oliver G, Sireau, Nicolas, Le Quan Sang, Kim-Hanh, Genovese, Federica, Braconi, Daniela, Santucci, Annalisa, Nemethova, Martina, Zatkova, Andrea, McCaffrey, Judith, Christensen, Peter, Ross, Gordon, Imrich, Richard, Rovensky, Jozef
Published in Annals of the rheumatic diseases (01.02.2016)
Published in Annals of the rheumatic diseases (01.02.2016)
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Efficacy and safety of once-daily nitisinone for patients with alkaptonuria (SONIA 2): an international, multicentre, open-label, randomised controlled trial
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Published in The lancet. Diabetes & endocrinology (01.09.2020)
Published in The lancet. Diabetes & endocrinology (01.09.2020)
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Homogentisic acid is not only eliminated by glomerular filtration and tubular secretion but also produced in the kidney in alkaptonuria
Ranganath, Lakshminarayan R., Milan, Anna M., Hughes, Andrew T., Khedr, Milad, Davison, Andrew S., Shweihdi, Ella, Norman, Brendan P., Hughes, Juliette H., Bygott, Helen, Luangrath, Emily, Fitzgerald, Richard, Psarelli, Eftychia E., Kan, Christa, Laan, Dinny, Olsson, Birgitta, Rudebeck, Mattias, Mankowitz, Louise, Sireau, Nicolas, Arnoux, Jean‐Baptiste, Le Quan Sang, Kim‐Hanh, Jarvis, Jonathan C., Genovese, Federica, Braconi, Daniela, Santucci, Annalisa, Zatkova, Andrea, Glasova, Helena, Stančík, Roman, Imrich, Richard, Rhodes, Nicholas P., Gallagher, James A.
Published in Journal of inherited metabolic disease (01.07.2020)
Published in Journal of inherited metabolic disease (01.07.2020)
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