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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
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
Sussman G, Abuzakouk M, Berard F, Canonica W, Oude Elberink H, Gimenez-Arnau A, Grattan C, Hollis K, Hunter S, Knulst A, Lacour JP, Lynde C, Marsland A, McBride D, Maurer M, Nakonechna A, Ortiz De Frutos J, Reynolds M, Sweeney C, Tian H, Weller K, Wolin D, Balp MM. Angioedema in chronic spontaneous urticaria is underdiagnosed and has a substantial impact: analyses from ASSURE-CSU. Allergy. 2018 Aug;73(8):1724-34. doi: 10.1111/all.13430
Maurer M, Abuzakouk M, Berard F, Canonica W, Oude Elberink H, Gimenez-Arnau A, Grattan C, Hollis K, Knulst A, Lacour JP, Lynde C, Marsland A, McBride D, Nakonechna A, Ortiz De Frutos J, Proctor C, Sussman G, Sweeney C, Tian H, Weller K, Wolin D, Balp M-M. The burden of chronic spontaneous urticaria is substantial: real-world evidence from ASSURE-CSU. Allergy. 2017 Dec;27(12):2005-16. doi: 10.1111/all.13209
Anto JM, Sunyer J, Basagana X, Garcia‐Esteban R, Cerveri I, de Marco R, Heinrich J, Janson C, Jarvis D, Kogevinas M, Kuenzli N, Leynaert B, Svanes C, Wjst M, Gislason T, Burney P. Risk factors of new-onset asthma in adults: a population-based international cohort study. Allergy. 2010 Aug;65(8):1021-30. doi: 10.1111/j.1398-9995.2009.02301.x