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Publications
Forsythe E, Haws RM, Argente J, Beales P, Martos-Moreno GA, Dollfus H, Chirila C, Gnanasakthy A, Buckley BC, Mallya UG, Clement K, Haqq AM. Quality of life improvements following one year of setmelanotide in children and adult patients with Bardet-Biedl syndrome: phase 3 trial results. Orphanet J Rare Dis. 2023 Jan 16;18(1):12. doi: 10.1186/s13023-022-02602-4
Kuhnen P, Wabitsch M, von Schnurbein J, Chirila C, Mallya UG, Callahan P, Gnanasakthy A, Poitou C, Krabusch PM, Stewart M, Clement K. Quality of life outcomes in two phase 3 trials of setmelanotide in patients with obesity due to LEPR or POMC deficiency. Orphanet J Rare Dis. 2022 Feb 5;17(1):38. doi: 10.1186/s13023-022-02186-z
Foy R, Willis T, Glidewell L, McEachan R, Lawton R, Meads D, Collinson M, Hunter C, Hulme C, West R, Ward V, Hartley S, Carder P, Alderson S, Holland M, Heudtlass P, Bregantini D, Schmitt L, Clamp S, Stokes T, Ingleson E, Rathfelder M, Johnson S, Richardson J, Rushforth B, Petty D, Vargas-Palacios A, Louch G, Heyhoe J, Watt I, Farrin A. Developing and evaluating packages to support implementation of quality indicators in general practice: the ASPIRE research programme, including two cluster RCTs. Programme Grants Appl Res. 2020 Mar;8(4). doi: 10.3310/pgfar08040
Noel E, Dussol B, Lacombe D, Bedreddine N, Fouilhoux A, Ronco P, Genevaz D, Bekri S, Hagege A, Dupuis-Simeon F, Derrien Ansquer V, Germain DP, Lidove O. Treatment needs and expectations for Fabry disease in France: development of a new Patient Needs Questionnaire. Orphanet J Rare Dis. 2019 Dec 4;14(1):284. doi: 10.1186/s13023-019-1254-7
Paquin RS, Fischer R, Mansfield C, Mange B, Beaverson K, Ganot A, Martin AS, Morris C, Rensch C, Ricotti V, Russo LJ, Sadosky A, Smith EC, Peay H. Priorities when deciding on participation in early-phase gene therapy trials for Duchenne muscular dystrophy: a best–worst scaling experiment in caregivers and adult patients. Orphanet J Rare Dis. 2019 May 9;14(1):102. doi: 10.1186/s13023-019-1069-6
Heller S, Lawton J, Amiel S, Cooke D, Mansell P, Brennan A, Elliott J, Boote J, Emery C, Baird W, Basarir H, Beveridge S, Bond R, Campbell M, Chater T, Choudhary P, Clark M, de Zoysa N, Dixon S, Gianfrancesco C, Hopkins D, Jacques R, Kruger J, Moore S, Oliver L, Peasgood T, Rankin D, Roberts S, Rogers H, Taylor C, Thokala P, Thompson G, Ward C. Improving management of type 1 diabetes in the UK: the Dose Adjustment For Normal Eating (DAFNE) programme as a research test-bed. A mixed-method analysis of the barriers to and facilitators of successful diabetes self-management, a health economic analysis, a cluster randomised controlled trial of different models of delivery of an educational intervention and the potential of insulin pumps and additional educator input to improve outcomes. Programme Grants Appl Res. 2014 Dec;2(5). doi: 10.3310/pgfar02050