1. Efficacy and safety of once-daily nitisinone for patients with alkaptonuria (SONIA 2): an international, multicentre, open-label, randomised controlled trial
- Author
-
Ranganath, Lakshminarayan R, Psarelli, Eftychia Eirini, Arnoux, Jean-Baptiste, Braconi, Daniela, Briggs, Michael, Bröijersén, Anders, Loftus, Nadia, Bygott, Helen, Cox, Trevor F, Davison, Andrew S, Dillon, Jane P, Fisher, Michael, FitzGerald, Richard, Genovese, Federica, Glasova, Helena, Hall, Anthony K, Hughes, Andrew T, Hughes, Juliette H, Imrich, Richard, Jarvis, Jonathan C, Khedr, Milad, Laan, Dinny, Le Quan Sang, Kim-Hanh, Luangrath, Emily, Lukáčová, Ol'ga, Milan, Anna M, Mistry, Alpesh, Mlynáriková, Vanda, Norman, Brendan P, Olsson, Birgitta, Rhodes, Nicholas P, Rovenský, Jozef, Rudebeck, Mattias, Santucci, Annalisa, Shweihdi, Ella, Scott, Ciarán, Sedláková, Jana, Sireau, Nicolas, Stančík, Roman, Szamosi, Johan, Taylor, Sophie, van Kan, Christa, Vinjamuri, Sobhan, Vrtíková, Eva, Webb, Chris, West, Elizabeth, Záňová, Elizabeth, Zatkova, Andrea, and Gallagher, James A
- Abstract
Alkaptonuria is a rare, genetic, multisystem disease characterised by the accumulation of homogentisic acid (HGA). No HGA-lowering therapy has been approved to date. The aim of SONIA 2 was to investigate the efficacy and safety of once-daily nitisinone for reducing HGA excretion in patients with alkaptonuria and to evaluate whether nitisinone has a clinical benefit.
- Published
- 2020
- Full Text
- View/download PDF