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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
Goldberg X, Castano-Vinyals G, Espinosa A, Carreras A, Liutsko L, Sicuri E, Foraster M , O'Callaghan-Gordo C, Dadvand P, Moncunill G, Dobano C, Cortes B, Pleguezuelos V, Straif K, Garcia-Aymerich J, de Cid R, Cardis E, Kogevinas M. Mental health and COVID-19 in a general population cohort in Spain (COVIVAT Study) . Soc Psychiatry Psychiatr Epidemiol. 2022 Dec;57(12):2457-68. doi: 10.1007/s00127-022-02303-0
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
Harries M, Mohr P, Grange F, Ehness R, Benjamin L, Siakpere O, Barth J, Stapelkamp C, Pfersch S, McLeod L , Wolowacz S , Kaye JA, Kontoudis I. Treatment patterns and outcomes of Stage IIIB/IIIC melanoma in France, Germany and the UK: a retrospective and prospective observational study (MELABIS) . Int J Clin Pract. 2017 May 16;71(5):e12946. doi: 10.1111/ijcp.12946