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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
Mareque M, Montesinos P, Font P, Guinea JM, de la Fuente A, Soto J, Oyaguez I, Brockbank J , Iglesias T, Llinares J, Sierra J. Cost-effectiveness analysis of gemtuzumab ozogamicin for first-line treatment of patients with Cd-33 positive acute myeloid leukaemia in Spain . Clinicoecon Outcomes Res. 2021 Apr 22;2021(13):263-77. doi: 10.2147/CEOR.S302097
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
Abdul-Jawad Altisent O, Puri R, Regueiro A, Chamandi C, Rodriguez-Gabella T, Del Trigo M, Campelo-Parada F, Couture T, Marsal JR , Cote M, Paradis JM, DeLarochellière R, Doyle D, Mohammadi S, Dumont E, Rodes-Cabau J. Predictors and association with clinical outcomes of the changes in exercise capacity after transcatheter aortic valve replacement . Circulation. 2017 Aug 15;136(7):632-43. doi: 10.1161/CIRCULATIONAHA.116.026349
Pladevall M , Brotons C, Gabriel R, Arnau A, Suarez C, de la Figuera M, Marquez E, Coca A, Sobrino J, Divine G, Heisler M, Williams LK, Writing Committee on behalf of COM99 Study Group . Multicenter cluster-randomized trial of a multifactorial intervention to improve antihypertensive medication adherence and blood pressure control among patients at high cardiovascular risk (The COM99 Study) . Circulation. 2010 Sep 21;122(12):1183-91.
Ferreira-Gonzalez I, Marsal JR , Ribera A, Permanyer-Miralda G, Garcia-Del Blanco B, Marti G, Cascant P, Martin-Yuste V, Brugaletta S, Sabate M, Alfonso F, Capote ML, De La Torre JM, Ruiz-Lera M, Sanmiguel D, Cardenas M, Pujol B, Baz JA, Iniguez A, Trillo R, Gonzalez-Bejar O, Casanova J, Sanchez-Gila J, Garcia-Dorado D. Background, incidence, and predictors of antiplatelet therapy discontinuation during the first year after drug-eluting stent implantation . Circulation. 2010 Sep 7;122(10):1017-25. doi: 10.1161/CIRCULATIONAHA.110.938290
Flavell DM, Jamshidi Y, Hawe E , Pineda Torra I, Taskinen MR, Frick MH, Nieminen MS, Kesäniemi YA, Pasternack A, Staels B, Miller G, Humphries SE, Talmud PJ, Syvänne M. Peroxisome proliferator-activated receptor alpha gene variants influence progression of coronary atherosclerosis and risk of coronary artery disease . Circulation. 2002 Feb 25;105(12):1440-5.