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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, Lynde C, Zanganeh S, Lacuesta G, Chiva-Razavi S, Chambenoit O, Khalil S, Hollis KA, McBride DW, Westlund RE, Sweeney CT, Tian H, Balp MM. ASSURE-CSU preliminary Canadian results: an analysis of the diagnosis and treatment of patients with antihistamine-refractory CSU. Poster presented at the 23rd World Congress of Dermatology; June 9, 2015. Vancouver, Canada. [abstract] Clin Transl Allergy. 2015 Aug 17; 5(29):1-7. doi: 10.1186/s13601-015-0072-9
Weller K, Maurer M, Grattan C, Nakonechna A, Abuzakouk M, Bérard F, Sussman G, Giménez-Arnau AM, Ortiz De Frutos J, Knulst A, Canonica GW, Hollis K, McBride D, Balp MM. ASSURE-CSU: a real-world study of burden of disease in patients with symptomatic chronic spontaneous urticaria. Clin Transl Allergy. 2015 Aug 17;17(5):29.