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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
Braitmaier M, Kollhorst B, Heinig M, Langner I, Czwikla J, Heinze F, Buschmann L, Minnerup H, Garcia-Albeniz X, Hense HW, Karch A, Zeeb H, Haug U, Didelez V. Effectiveness of mammography screening on breast cancer mortality - a study protocol for emulation of target trials using German health claims data. Clin Epidemiol. 2022 Nov 2;14:1293-303. doi: 10.2147/CLEP.S376107
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
Arana A, Pottegard A, Kuiper J, Booth H, Reutfors J, Calingaert B, Lund LC, Crellin E, Schmitt-Egenolf M, Kaye J, Gembert K, Rothman K, Kieler H, Dedman D, Houben E, Gutierrez L, Hallas J, Perez-Gutthann S. Long-term risk of skin cancer and lymphoma in users of topical tacrolimus and pimecrolimus: final results from the extension of the cohort study Protopic Joint European Longitudinal Lymphoma and Skin Cancer Evaluation (JOELLE). Clin Epidemiol. 2021 Dec 29;2021(13):1141-53. doi: 10.2147/CLEP.S331287
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
Anthony MS, Armstrong MA, Getahun D, Scholes D, Gatz J, Schulze-Rath R, Postlethwaite D, Merchant M, Alabaster AL, Chillemi G, Raine-Bennett T, Xie F, Chiu VY, Im TM, Takhar HS, Fassett M, Grafton J, Cronkite D, Ichikawa L, Reed SD, Hui SL, Ritchey ME, Saltus CW, Andrews EB, Rothman KJ, Asiimwe A, Lynen R, Schoendorf J. Identification and validation of uterine perforation, intrauterine device expulsion, and breastfeeding in four health care systems with electronic health records. Clin Epidemiol. 2019 Jul 23;2019(11):635-43. doi: 10.2147/CLEP.S201044
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
Castellsague J, Kuiper JG, Pottegard A, Anveden Berglind I, Dedman D, Gutierrez L, Calingaert B, van Herk-Sukel MP, Hallas J, Sundstrom A, Gallagher AM, Kaye JA, Pardo C, Rothman KJ, Perez-Gutthann S. A cohort study on the risk of lymphoma and skin cancer in users of topical tacrolimus, pimecrolimus, and corticosteroids (Joint European Longitudinal Lymphoma and Skin Cancer Evaluation – JOELLE study). Clin Epidemiol. 2018 Mar 13;2018(10):299-310. doi: 10.2147/CLEP.S146442