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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
Richardson D , Zhan L, Mahtani R, McRoy L, Mitra D, Reynolds M , Odom D , Hollis K , Kaye JA, Jones C, Hargis J. A prospective observational study of patient-reported functioning and quality of life in advanced and metastatic breast cancer utilizing a novel mobile application . Breast Cancer Res Treat. 2021 May;187(1):113-24. doi: 10.1007/s10549-020-06082-7
Stenehjem DD, Telford C, Unni SK, Bauer H, Sainski A, Deka R, Schauerhamer MB, Ye X, Tak CR, Ma J, Dalvi TB, Gutierrez L , Kaye JA, Tyczynski JE, Brixner DI, Biskupiak JE. BRCA testing and outcomes in women with breast cancer . Breast Cancer Res Treat. 2021 Apr;186(3):839-50. doi: 10.1007/s10549-020-06038-x
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
Liede A, Mansfield CA , Metcalfe KA, Price MA, Kathleen Cuningham Foundation Consortium for Research into Familial Breast Cancer, Snyder C, Lynch HT, Friedman S, Amelio J, Posner J, Narod SA, Lindeman GJ, Evans DG. Preferences for breast cancer risk reduction among BRCA1/BRCA2 mutation carriers: a discrete-choice experiment . Breast Cancer Res Treat. 2017 Sep;165(2):433-44. doi: 10.1007/s10549-017-4332-3.
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
Dine JL , O'Sullivan CC, Voeller D, Greer YE, Chavez KJ, Conway CM, Sinclair S, Stone B, Amiri-Kordestani L, Merchant AS, Hewitt SM, Steinberg SM, Swain SM, Lipkowitz S. The TRAIL receptor agonist drozitumab targets basal B triple-negative breast cancer cells that express vimentin and Axl . Breast Cancer Res Treat. 2016 Jan;155(2):235-51. doi: 10.1007/s10549-015-3673-z.