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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).
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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)
(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.