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

Lakshminarayan R Ranganath; Anna M Milan; Andrew T Hughes; John J Dutton; Richard Fitzgerald; Michael C Briggs; Helen Bygott; Eftychia E Psarelli; Trevor F Cox; James A Gallagher; Jonathan C Jarvis; Christa van Kan; Anthony K Hall; Dinny Laan; Birgitta Olsson; Johan Szamosi; Mattias Rudebeck; Torbjörn Kullenberg; Arvid Cronlund; Lennart Svensson; Carin Junestrand; Hana Ayoob; Oliver G Timmis; Nicolas Sireau; Kim-Hanh Le Quan Sang; Federica Genovese; Daniela Braconi; Annalisa Santucci; Martina Nemethova; Andrea Zatkova; Judith McCaffrey; Peter Christensen; Gordon Ross; Richard Imrich; Jozef Rovensky

doi:10.1136/annrheumdis-2014-206033

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

Lakshminarayan R Ranganath, Anna M Milan, Andrew T Hughes, John J Dutton, Richard Fitzgerald, Michael C Briggs, Helen Bygott, Eftychia E Psarelli, Trevor F Cox, James A Gallagher, Jonathan C Jarvis, Christa van Kan, Anthony K Hall, Dinny Laan, Birgitta Olsson, Johan Szamosi, Mattias Rudebeck, Torbjörn Kullenberg, Arvid Cronlund, Lennart SvenssonCarin Junestrand, Hana Ayoob, Oliver G Timmis, Nicolas Sireau, Kim-Hanh Le Quan Sang, Federica Genovese, Daniela Braconi, Annalisa Santucci, Martina Nemethova, Andrea Zatkova, Judith McCaffrey, Peter Christensen, Gordon Ross, Richard Imrich, Jozef Rovensky

Norwich Medical School

Research output: Contribution to journal › Article › peer-review

115 Citations (SciVal)

Abstract

Alkaptonuria (AKU) is a serious genetic disease characterised by premature spondyloarthropathy. Homogentisate-lowering therapy is being investigated for AKU. Nitisinone decreases homogentisic acid (HGA) in AKU but the dose-response relationship has not been previously studied.

Original language	English
Pages (from-to)	362-367
Journal	Annals of the Rheumatic Diseases
Volume	75
Issue number	2
Early online date	4 Dec 2014
DOIs	https://doi.org/10.1136/annrheumdis-2014-206033
Publication status	Published - Feb 2016

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10.1136/annrheumdis-2014-206033

Cite this

Ranganath, L. R., Milan, A. M., Hughes, A. T., Dutton, J. J., Fitzgerald, R., Briggs, M. C., Bygott, H., Psarelli, E. E., Cox, T. F., Gallagher, J. A., Jarvis, J. C., van Kan, C., Hall, A. K., Laan, D., Olsson, B., Szamosi, J., Rudebeck, M., Kullenberg, T., Cronlund, A., ... Rovensky, J. (2016). 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. Annals of the Rheumatic Diseases, 75(2), 362-367. https://doi.org/10.1136/annrheumdis-2014-206033

Ranganath, Lakshminarayan R ; Milan, Anna M ; Hughes, Andrew T et al. / 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. In: Annals of the Rheumatic Diseases. 2016 ; Vol. 75, No. 2. pp. 362-367.

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title = "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",

abstract = "Alkaptonuria (AKU) is a serious genetic disease characterised by premature spondyloarthropathy. Homogentisate-lowering therapy is being investigated for AKU. Nitisinone decreases homogentisic acid (HGA) in AKU but the dose-response relationship has not been previously studied.",

author = "Ranganath, {Lakshminarayan R} and Milan, {Anna M} and Hughes, {Andrew T} and Dutton, {John J} and Richard Fitzgerald and Briggs, {Michael C} and Helen Bygott and Psarelli, {Eftychia E} and Cox, {Trevor F} and Gallagher, {James A} and Jarvis, {Jonathan C} and {van Kan}, Christa and Hall, {Anthony K} and Dinny Laan and Birgitta Olsson and Johan Szamosi and Mattias Rudebeck and Torbj{\"o}rn Kullenberg and Arvid Cronlund and Lennart Svensson and Carin Junestrand and Hana Ayoob and Timmis, {Oliver G} and Nicolas Sireau and {Le Quan Sang}, Kim-Hanh and Federica Genovese and Daniela Braconi and Annalisa Santucci and Martina Nemethova and Andrea Zatkova and Judith McCaffrey and Peter Christensen and Gordon Ross and Richard Imrich and Jozef Rovensky",

note = "Published by the BMJ Publishing Group Limited. For permission to use (where not already granted under a licence) please go to http://group.bmj.com/group/rights-licensing/permissions.",

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doi = "10.1136/annrheumdis-2014-206033",

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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, K-H, Genovese, F, Braconi, D, Santucci, A, Nemethova, M, Zatkova, A, McCaffrey, J, Christensen, P, Ross, G, Imrich, R & Rovensky, J 2016, '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', Annals of the Rheumatic Diseases, vol. 75, no. 2, pp. 362-367. https://doi.org/10.1136/annrheumdis-2014-206033

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 et al.

In: Annals of the Rheumatic Diseases, Vol. 75, No. 2, 02.2016, p. 362-367.

Research output: Contribution to journal › Article › peer-review

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AU - Ross, Gordon

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AB - Alkaptonuria (AKU) is a serious genetic disease characterised by premature spondyloarthropathy. Homogentisate-lowering therapy is being investigated for AKU. Nitisinone decreases homogentisic acid (HGA) in AKU but the dose-response relationship has not been previously studied.

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Ranganath LR, Milan AM, Hughes AT, Dutton JJ, Fitzgerald R, Briggs MC et al. 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. Annals of the Rheumatic Diseases. 2016 Feb;75(2):362-367. Epub 2014 Dec 4. doi: 10.1136/annrheumdis-2014-206033