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.

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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.

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Author and article information

Journal

Journal ID (iso-abbrev): Ann. Rheum. Dis.

Title: Annals of the rheumatic diseases

Publisher: BMJ

ISSN (Electronic): 1468-2060

ISSN (Print): 0003-4967

Publication date (Electronic): Feb 2016

Volume: 75

Issue: 2

Affiliations

[1 ] Department of Clinical Biochemistry and Metabolism, Royal Liverpool University Hospital, Liverpool, UK Department of Musculoskeletal Biology, University of Liverpool, Liverpool, UK.

[2 ] Department of Clinical Biochemistry and Metabolism, Royal Liverpool University Hospital, Liverpool, UK.

[3 ] Department of Clinical Pharmacology, Royal Liverpool University Hospital, Liverpool, UK.

[4 ] Department of Ophthalmology, Royal Liverpool University Hospital, Liverpool, UK.

[5 ] Department of Musculoskeletal Biology, Institute of Ageing and Chronic Disease, University of Liverpool, Liverpool, UK.

[6 ] Department of Musculoskeletal Biology, University of Liverpool, Liverpool, UK.

[7 ] School of Sport and Exercise Science, Liverpool John Moores University, Liverpool, UK.

[8 ] PSR group B.V., Hoofddorp, Netherlands.

[9 ] Cudos BV, Hoofddorp, Netherlands.

[10 ] Swedish Orphan Biovitrum AB (publ), Stockholm, Sweden.

[11 ] AKU Society, Cambridge, UK.

[12 ] Hôpital Necker-Enfants Malades, Paris Cedex 15, France.

[13 ] Nordic Bioscience, Herlev, Denmark.

[14 ] Dipartimento di Biotecnologie, Chimica e Farmacia, Università degli Studi di Siena, Siena, Italy.

[15 ] Laboratory of Genetics, Institute of Molecular Physiology and Genetics, Slovak Academy of Sciences, Bratislava, Slovakia.

[16 ] Agilent Technologies Ireland, Cork, Ireland.

[17 ] Agilent Technologies, Stockport, UK.

[18 ] Center for Molecular Medicine, Slovak Academy of Sciences, Bratislava, Slovakia.

[19 ] National Institute of Rheumatic Diseases, Piešťany, Slovakia.

Article

Publisher Item ID: annrheumdis-2014-206033

DOI: 10.1136/annrheumdis-2014-206033

PubMed ID: 25475116

SO-VID: b234af81-1330-44cc-a001-7622e015fcd5

History

Keywords: Arthritis,Osteoarthritis,Spondyloarthritis

Data availability:

Keywords: Arthritis, Osteoarthritis, Spondyloarthritis

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