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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
van Kamp I, Waye KP, Kanninen K, Gulliver J, Bozzon A, Psyllidis A, Boshuizen H, Selander J, van den Hazel P, Brambilla M, Foraster M , Julvez J, Klatte M, Jeram S, Lercher P, Botteldooren D, Ristovska G, Kaprio J, Schreckenberg D, Hornikx M, Fels J, Weber M, Braat-Eggen E, Hartmann J, Clark C, Vrijkotte T, Brown L, Bolte G. Early environmental quality and life-course mental health effects: the equal-life project . Environ Epidemiol. 2021 Dec 16;6(1):e183. doi: 10.1097/EE9.0000000000000183
Wesselink AK, Kirwa K, Hatch EE, Hystad P, Szpiro AA, Kaufman JD, Levy JI, Mikkelsen EM, Quraishi SM, Rothman KJ , Wise LA. Residential proximity to major roads and fecundability in a preconception cohort . Environ Epidemiol. 2020 Nov 11;4(6):e112. doi: 10.1097/EE9.0000000000000112
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
Nastoupil LJ, Sinha R, Byrtek M, Ziemiecki R , Zhou X , Taylor M, Friedberg JW, Link BK, Cerhan JR, Dawson K, Flowers CR. Outcomes following watchful waiting for stage II–IV follicular lymphoma patients in the modern era . Br J Haematol. 2016 Mar;172(5):724-34. doi: 10.1111/bjh.13895
Nabhan C, Byrtek M, Rai A, Dawson K, Zhou X , Link BK, Friedberg JW, Zelenetz AD, Maurer MJ, Cerhan JR, Flowers CR. Disease characteristics, treatment patterns, prognosis, outcomes and lymphoma-related mortality in elderly follicular lymphoma in the United States . Br J Haematol. 2015 Jul;170(1):85-95. doi: 10.1111/bjh.13399
Harrison CN, Mesa RA, Kiladjian J-J, Al-Ali H-K, Gisslinger H, Knoops L, Squier M, Sirulnik A, Mendelson E, Zhou X , Copley-Merriman C , Hunter DS, Levy RS, Cervantes F, Passamonti F, Barbui T, Barosi G, Vannucchi AM. Health-related quality of life and symptoms in patients with myelofibrosis treated with ruxolitinib versus best available therapy . Br J Haematol. 2013 Jul;162(2):229-39. doi: 10.1111/bjh.12375