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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
Wesselink AK, Wang TR, Ketzel M, Mikkelsen EM, Brandt J, Khan J, Hertel O, Laursen ASD, Johannesen BR, Willis MD, Levy JI, Rothman KJ, Sorensen HT, Wise LA, Hatch EE. Air pollution and fecundability: results from a Danish preconception cohort study. Paediatr Perinat Epidemiol. 2022 Jan;36(1):57-67. doi: 10.1111/ppe.12832
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
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
Wise LA, Rothman KJ, Mikkelsen EM, Stanford JB, Wesselink AK, McKinnon C, Gruschow SM, Horgan CE, Wiley AS, Hahn KA, Sørensen HT, Hatch EE. Design and conduct of an internet-based preconception cohort study in North America: pregnancy study online. Paediatr Perinat Epidemiol. 2015 Jul;29(4):360-71. doi: 10.1111/ppe.12201
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