Developing a cure for Black Bone Disease by Sireau Nicolas T

Clinical development innovation in rare diseases: overcoming barriers to successful delivery of a randomised clinical trial in alkaptonuria—a mini-review by L. R. Ranganath, Nick Sireau

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A patient survey on the impact of alkaptonuria symptoms as perceived by the patients and their experiences of receiving diagnosis and care by Mattias Rudebeck, Ciarán Scott, Nicolas Sireau, Lakshminarayan Ranganath

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A cost-of-illness analysis of the economic burden of obsessive-compulsive disorder in the United Kingdom by Naman Kochar, Sophie Ip, Vittoria Vardanega, Nick T. Sireau, Naomi A. Fineberg

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First decade anniversary of the United Kingdom National Alkaptonuria Centre by Milad Khedr, Nick Sireau, George Bou‐Gharios, Jim A. Gallagher, Lakshminarayan R. Ranganath

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Evaluation of Homogentisic Acid, a Prospective Antibacterial Agent Highlighted by the Suitability of Nitisinone in Alkaptonuria 2 (SONIA 2) Clinical Trial by Nicola Ooi, Ian R. Cooper, Brendan Norman, James A. Gallagher, Nick Sireau, George Bou-Gharios, Lakshminarayan R. Ranganath, Victoria J. Savage

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Clinical development innovation in rare diseases: lessons learned and best practices from the DevelopAKUre consortium by Mattias Rudebeck, Ciarán Scott, Nicholas P. Rhodes, Christa van Kan, Birgitta Olsson, Mohammed Al-sbou, Anthony K. Hall, Nicolas Sireau, Lakshminarayan R. Ranganath

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3D printed customised external cranial plate in a patient with syndrome of trephined: ‘a case report’ by Mee H., Greasley S., Whiting G., Harkin C., Oliver G., Marsden D., Andrews R., Sireau S., Price RD., Anwar F., Timofeev IS., Hutchinson PJ., White PA., Helmy A.

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Comparing nitisinone 2 mg and 10 mg in the treatment of alkaptonuria—An approach using statistical modelling by Lakshminarayan R. Ranganath, Anna M. Milan, Andrew T. Hughes, Milad Khedr, Brendan P. Norman, Mohammed Alsbou, Richard Imrich, Matthew Gornall, Nicolas Sireau, James A. Gallagher, Richard Jackson

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Identifying obstacles hindering the conduct of academic-sponsored trials for drug repurposing on rare-diseases: an analysis of six use cases by Marta del Álamo, Christoph Bührer, Dirk Fisher, Matthias Griese, Paul Lingor, Giovanni Palladini, Nicolas Sireau, Virginie Hivert, Luca Sangiorgi, Florence Guillot, Juliane Halftermeyer, Lenka Soucková, Kristýna Nosková, Regina Demlová

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Feasibility and acceptability of transcranial stimulation in obsessive–compulsive symptoms (FEATSOCS): study protocol for a randomised controlled trial of transcranial direct curre... by Eduardo Cinosi, David Adam, Ibrahim Aslan, David Baldwin, Kieran Chillingsworth, Arun Enara, Tim Gale, Kabir Garg, Matthew Garner, Robert Gordon, Natalie Hall, Nathan T. M. Huneke, Sonay Kucukterzi-Ali, Joanne McCarthy, Daniel Meron, Deela Monji-Patel, Roisin Mooney, Trevor Robbins, Megan Smith, Nick Sireau, David Wellsted, Solange Wyatt, Naomi A. Fineberg

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How to manage obsessive-compulsive disorder (OCD) under COVID-19: A clinician's guide from the International College of Obsessive Compulsive Spectrum Disorders (ICOCS) and the Obse... by N.A. Fineberg, M. Van Ameringen, L. Drummond, E. Hollander, D.J. Stein, D. Geller, S. Walitza, S. Pallanti, L. Pellegrini, J. Zohar, C.I. Rodriguez, J.M. Menchon, P. Morgado, D. Mpavaenda, L.F. Fontenelle, J.D. Feusner, G. Grassi, C. Lochner, D.J. Veltman, N. Sireau, L. Carmi, D. Adam, H. Nicolini, B. Dell'Osso

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Data on items of AKUSSI in Alkaptonuria collected over three years from the United Kingdom National Alkaptonuria Centre and the impact of nitisinone by R. Griffin, E.E. Psarelli, T.F. Cox, M. Khedr, A.M. Milan, A.S. Davison, A.T. Hughes, J.L. Usher, S. Taylor, N. Loftus, A. Daroszewska, E. West, A. Jones, M. Briggs, M. Fisher, M. McCormick, S. Judd, S. Vinjamuri, N. Sireau, J.P. Dillon, J.M. Devine, G. Hughes, J. Harrold, G.J. Barton, J.C. Jarvis, J.A. Gallagher, L.R. Ranganath

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