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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
Laurita Longo V, Odjidja EN, Beia TK, Neri M, Kielmann K, Gittardi I, Di Rosa AI, Boldrini M, Melis GB, Scambia G, Lanzone A. "An unnecessary cut?" multilevel health systems analysis of drivers of caesarean sections rates in Italy: a systematic review. BMC Pregnancy Childbirth. 2020 Dec 10;20(1):770. doi: 10.1186/s12884-020-03462-1
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
Jimenez-Murcia S, Granero R, Larrea ST, Sauvaget A, Grall-Bronnec M, Alvarez-Moya EM, Aguera Z, Aymami N, Gomez-Pena M, Del Pino-Gutierrez A, Moragas L, Menchon JM, Fagundo AB, Sauchelli S, La Verde M, Aguglia E, Signorelli MS, Formoso JAF, Fernandez-Aranda F. Pathological gambling and substance use disorders: effects of early incidence and personality. Drugs Health Society. 2015 Jan;14(1):78. doi: 10.7202/1035550ar