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Publications
Melen E, Faner R, Allinson JP, Bui D, Bush A, Custovic A, Garcia-Aymerich J, Guerra S, Breyer-Kohansal R, Hallberg J, Lahousse L, Martinez FD, Merid SK, Powell P, Pinnock H, Stanojevic S, Vanfleteren LEGW, Wang G, Dharmage SC, Wedzicha J, Agusti Alvar, Abellan A , CADSET Investigators. Lung-function trajectories: relevance and implementation in clinical practice . Lancet. 2024 Apr 13;403(10435):1494-503. doi: 10.1016/S0140-6736(24)00016-3
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
Reed SD, Zhou X , Ichikawa L, Gatz JL, Peipert JF, Armstrong MA, Raine-Bennett T, Getahun D, Fassett MJ, Postlethwaite DA, Shi JM, Asiimwe A, Pisa F, Schoendorf J, Saltus CW , Anthony MS , APEX-IUD study team. Intrauterine device-related uterine perforation incidence and risk (APEX-IUD): a large multisite cohort study . Lancet. 2022 Jun 4;399(10341):2103-12. doi: 10.1016/S0140-6736(22)00015-0
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
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
Fine P, Victora CG, Rothman KJ , Moore PS, Chang Y, Curtis V, Heymann DL, Slutkin G, May RM, Patel V, Roberts I, Wortley R, Torgerson C, Deaton A. John Snow's legacy: epidemiology without borders . Lancet. 2013 Apr 1;381(9874):1302-11.