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

Author:

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

Publisher

BMJ

Subject

General Biochemistry, Genetics and Molecular Biology,Immunology,Immunology and Allergy,Rheumatology

Reference17 articles.

1. Alkaptonuria—a review of surgical and autopsy pathology;Helliwell;Histopath,2008

2. Biochemical, pathologic and clinical aspects of alcaptonuria, ochronosis and ochronotic arthropathy: review of world literature (1584–1962);O'Brien;Am J Med,1963

3. Natural history of alkaptonuria revisited: analyses based on scoring systems;Ranganath;J Inherit Metab Dis,2011

4. Natural history of Alkaptonuria;Phornphutkul;N Engl J Med,2002

5. An update on molecular genetics of Alkaptonuria (AKU);Zatkova;J Inherit Metab Dis,2011

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