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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.