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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
Mazurek M, Halperin JL, Huisman MV, Diener HC, Dubner SJ, Ma CS, Rothman KJ, Healey JS, Teutsch C, Paquette M, Franca LR, Lu S, Bartels DB, Lip GYH. Antithrombotic treatment for newly diagnosed atrial fibrillation in relation to patient age: the GLORIA-AF registry programme. Europace. 2020 Jan 1;22(1):47-57. doi: 10.1093/europace/euz278
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
Vinceti M, Filippini T, Violi F, Rothman KJ, Costanzini S, Malagoli C, Wise LA, Odone A, Signorelli C, Iacuzio L, Arcolin E, Mandrioli J, Fini N, Patti F, Lo Fermo S, Pietrini V, Teggi S, Ghermandi G, Scillieri R, Ledda C, Mauceri C, Sciacca S, Fiore M, Ferrante M. Pesticide exposure assessed through agricultural crop proximity and risk of amyotrophic lateral sclerosis. Environ Health. 2017 Aug 29;16(1):91. doi: 10.1186/s12940-017-0297-2
Huisman MV, Ma CS, Diener HC, Dubner SJ, Halperin JL, Rothman KJ, Teutsch C, Schoof N, Kleine E, Bartels DB, Lip GY. Antithrombotic therapy use in patients with atrial fibrillation before the era of non-vitamin K antagonist oral anticoagulants: the Global Registry on Long-Term Oral Antithrombotic Treatment in Patients with Atrial Fibrillation (GLORIA-AF) phase I cohort. Europace. 2016 Sep;18(9):1308-18. doi: 10.1093/europace/euw073
Casas L, Tischer C, Wouters IM, Torrent M, Gehring U, Garcia-Esteban R, Thiering E, Postma DS, de Jongste J, Smit HA, Borras-Santos A, Zock JP, Hyvarinen A, Heinrich J, Sunyer J. Early life microbial exposure and fractional exhaled nitric oxide in school-age children: a prospective birth cohort study. Environ Health. 2013 Dec 2;12:103. doi: 10.1186/1476-069X-12-103