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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
Hunter JE, Arnold KA, Cook JE, Zepp J, Gilmore MJ, Rope AF, Davis JV, Bergen KM, Esterberg E, Muessig KR, Peterson SK, Syngal S, Acheson L, Wiesner G, Reiss J, Goddard KAB. Universal screening for Lynch syndrome among patients with colorectal cancer: patient perspectives on screening and sharing results with at-risk relatives. Fam Cancer. 2017 Jul;16(3):377-87. doi: 10.1007/s10689-017-9972-2
Hunter JE, Zepp JM, Gilmore MJ, Davis JV, Esterberg EJ, Muessig KR, Peterson SK, Syngal S, Acheson LS, Wiesner GL, Reiss JA, Goddard KA. Universal tumor screening for Lynch syndrome: assessment of the perspectives of patients with colorectal cancer regarding benefits and barriers. Cancer. 2015 Sep 15;121(18):3281-9. doi: 10.1002/cncr.29470
Aguilera I, Foraster M, Basagana X, Corradi E, Deltell A, Morelli X, Phuleria HC, Ragettli MS, Rivera M, Thomasson A, Slama R, Kunzli N. Application of land use regression modelling to assess the spatial distribution of road traffic noise in three European cities. J Expo Sci Environ Epidemiol. 2015 Jan;25(1):97-105. doi: 10.1038/jes.2014.61
Burris HA, Lebrun F, Rugo HS, Beck T, Piccart M, Neven P, Baselga J, Petrakova K, Hortobagyi GN, Komorowski A, Chouinard E, Young R, Gnant M, Pritchard KI, Bennett L, Ricci J-F, Bauly H, Taran T, Sahmoud T, Noguchi S. Health-related quality of life of patients with advanced breast cancer treated with everolimus plus exemestane versus placebo plus exemestane in the phase 3, randomized, controlled, BOLERO-2 trial. Cancer. 2013 Mar 15;119(10):1908-15.
Maurel J, Martins AS, Poveda A, Lopez-Guerrero JA, Cubedo R, Casado A, Martinez-Trufero J, Ramon Ayuso J, Lopez-Pousa A, Garcia-Albeniz X, Garcia del Muro X, de Alava E. Imatinib plus low-dose doxorubicin in patients with advanced gastrointestinal stromal tumors refractory to high-dose imatinib: a phase I-II study by the Spanish Group for Research on Sarcomas. Cancer. 2010 Aug 1;116(15):3692-701. doi: 10.1002/cncr.25111