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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
DiBenedetti D, Slota C, Wronski SL, Vradenburg G, Comer M, Callahan LF, Winfield J, Rubino I, Krasa HB, Hartry A, Wieberg D, Kremer IN, Lappin D, Martin AD, Frangiosa T, Biggar V, Hauber B. Assessing what matters most to patients with or at risk for Alzheimer's and care partners: a qualitative study evaluating symptoms, impacts, and outcomes. Alzheimers Res Ther. 2020 Jul 30;12(90). doi: 10.1186/s13195-020-00659-6
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
Vinceti M, Chiari A, Eichmuller M, Rothman KJ, Filippini T, Malagoli C, Weuve J, Tondelli M, Zamboni G, Nichelli PF, Michalke B. A selenium species in cerebrospinal fluid predicts conversion to Alzheimer's dementia in persons with mild cognitive impairment. Alzheimers Res Ther. 2017 Dec 19;9(1):100. doi: 10.1186/s13195-017-0323-1
Archer R, Tappenden P, Ren S, Martyn-St James M, Harvey R, Basarir H, Stevens J, Carroll C, Cantrell A, Lobo A, Hoque S. Infliximab, adalimumab and golimumab for treating moderately to severely active ulcerative colitis after the failure of conventional therapy (including a review of TA140 and TA262): clinical effectiveness systematic review and economic model. Health Technol Assess. 2016 May;20(39):1-326. doi: 10.3310/hta20390
Olson J, Sharp P, Goatman K, Prescott G, Scotland G, Fleming A, Philip S, Santiago C, Borooah S, Broadbent D, Chong V, Dodson P, Harding S, Leese G, Styles C, Swa K, Wharton H. Improving the economic value of photographic screening for optical coherence tomography-detectable macular oedema: a prospective, multicentre, UK study. Health Technol Assess. 2013 Nov 1;17(51):1-142. doi: 10.3310/hta17510