Rheumatology, Osteoarthritis
4. Dec 2014
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
BACKGROUND:
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.
METHOD…
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Authors:
- 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,