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Rudebeck, Mattias, Scott, Ciaran, Rhodes, Nicholas P ORCID: 0000-0002-5173-7032, van Kan, Christa, Olsson, Birgitta, Al-sbou, Mohammed, Hall, Anthony K, Sireau, Nicolas and Ranganath, Lakshminarayan R (2021) Clinical development innovation in rare diseases: lessons learned and best practices from the DevelopAKUre consortium. ORPHANET JOURNAL OF RARE DISEASES, 16 (1). 510-.

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Ranganath, Lakshminarayan R, Psarelli, Eftychia Eirini ORCID: 0000-0002-3102-0288, Arnoux, Jean-Baptiste, Braconi, Daniela, Briggs, Michael, Broijersen, Anders, Loftus, Nadia, Bygott, Helen, Cox, Trevor F, Davison, Andrew S ORCID: 0000-0001-5501-4475 et al (show 39 more authors) , Dillon, Jane P ORCID: 0000-0002-7055-5664, Fisher, Michael, FitzGerald, Richard, Genovese, Federica, Glasova, Helena, Hall, Anthony K, Hughes, Andrew T, Hughes, Juliette H ORCID: 0000-0001-6155-4136, Imrich, Richard, Jarvis, Jonathan C, Khedr, Milad, Laan, Dinny, Sang, Kim-Hanh Le Quan, Luangrath, Emily, Lukacova, Olga, Milan, Anna M, Mistry, Alpesh, Mlynarikova, Vanda, Norman, Brendan P ORCID: 0000-0001-9293-4852, Olsson, Birgitta, Rhodes, Nicholas P ORCID: 0000-0002-5173-7032, Rovensky, Jozef, Rudebeck, Mattias, Santucci, Annalisa, Shweihdi, Ella, Scott, Ciaran, Sedlakova, Jana, Sireau, Nicolas, Stancik, Roman, Szamosi, Johan, Taylor, Sophie, van Kan, Christa, Vinjamuri, Sobhan, Vrtikova, Eva, Webb, Chris, West, Elizabeth, Zanova, Elizabeth, Zatkova, Andrea and Gallagher, James A ORCID: 0000-0002-0852-279X (2020) Efficacy and safety of once-daily nitisinone for patients with alkaptonuria (SONIA 2): an international, multicentre, open-label, randomised controlled trial. The Lancet Diabetes and Endocrinology, 8 (9). pp. 762-772.

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Ranganath, Lakshminarayan R, Milan, Anna M ORCID: 0000-0002-0452-2338, Hughes, Andrew T, Khedr, Milad ORCID: 0000-0002-4998-2397, Davison, Andrew S ORCID: 0000-0001-5501-4475, Shweihdi, Ella, Norman, Brendan P ORCID: 0000-0001-9293-4852, Hughes, Juliette H ORCID: 0000-0001-6155-4136, Bygott, Helen, Luangrath, Emily et al (show 20 more authors) , Fitzgerald, Richard, Psarelli, Eftychia E ORCID: 0000-0002-3102-0288, van Kan, Christa, Laan, Dinny, Olsson, Birgitta, Rudebeck, Mattias, Mankowitz, Louise, Sireau, Nicolas, Arnoux, Jean-Baptiste, Sang, Kim-Hanh Le Quan, Jarvis, Jonathan C, Genovese, Federica, Braconi, Daniela, Santucci, Annalisa, Zatkova, Andrea, Glasova, Helena, Stancik, Roman, Imrich, Richard, Rhodes, Nicholas P ORCID: 0000-0002-5173-7032 and Gallagher, James A ORCID: 0000-0002-0852-279X (2020) Homogentisic acid is not only eliminated by glomerular filtration and tubular secretion but also produced in the kidney in alkaptonuria. JOURNAL OF INHERITED METABOLIC DISEASE, 43 (4). pp. 737-747.