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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
Schjesvold F, Goldschmidt H, Maisnar V, Spicka I, Abildgaard N, Rowlings P, Cain L, Romanus D, Suryanarayan K, Rajkumar V, Odom D, Gnanasakthy A, Dimopoulos MA. Quality of life is maintained with ixazomib maintenance in post-transplant newly diagnosed multiple myeloma: the TOURMALINE-MM3 trial. Eur J Haematol. 2020 May;104(5):443-58. doi: 10.1111/ejh.13379.
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
Mazurek M, Teutsch C, Diener H-C, Dubner SJ, Halperin JL, Ma C-S, Rothman KJ, Paquette M, Zint K, Franca L, Lu S, Bartels DB, Huisman MV, Lip GYH, GLORIA-AF Investigators. Safety and effectiveness of dabigatran at 2 years: final outcomes from Phase II of the GLORIA-AF registry program. Am Heart J. 2019 Dec;218:123-7. doi: 10.1016/j.ahj.2019.08.012
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
Cainzos-Achirica M, Rebordosa C, Vela E, Cleries M, Matsushita K, Plana E, Rivero-Ferrer E, Enjuanes C, Jimenez-Marrero S, Garcia-Rodriguez LA, Comin-Colet J, Perez-Gutthann S. Challenges of evaluating chronic heart failure and acute heart failure events in research studies using large health care databases. Am Heart J. 2018 Aug;202:76-83. doi: 10.1016/j.ahj.2018.05.005
Blonde L, Raccah D, Lew E, Meyers J, Nikonova E, Ajmera M, Davis KL, Bertolini M, Guerci B. Treatment intensification in type 2 diabetes: a real world study of 2-OAD regimens, GLP-1 RAx, or basal insulin. Diabetes Ther. 2018 Jun;9(3):1169-84. doi: 10.1007/s13300-018-0429-x
Huisman MV, Lip GY, Diener HC, Dubner SJ, Halperin JL, Ma CS, Rothman KJ, Teutsch C, Zint K, Ackermann D, Clemens A, Bartels DB. Design and rationale of global registry on long-term oral antithrombotic treatment in patients with atrial fibrillation: a global registry program on long-term oral antithrombotic treatment in patients with atrial fibrillation. Am Heart J. 2014 Mar;167(3):329-34. doi: 10.1016/j.ahj.2013.12.006
Abu-Assi E, Ferreira-Gonzalez I, Ribera A, Marsal JR, Cascant P, Heras M, Bueno H, Sanchez PL, Aros F, Marrugat J, Garcia-Dorado D, Pena-Gil C, Gonzalez-Juanatey JR, Permanyer-Miralda G. Do GRACE (Global Registry of Acute Coronary events) risk scores still maintain their performance for predicting mortality in the era of contemporary management of acute coronary syndromes? Am Heart J. 2010 Nov;160(5):826-34. doi: 10.1016/j.ahj.2010.06.053
Ferreira-Gonzalez I, Permanyer-Miralda G, Heras M, Cunat J, Civeira E, Aros F, Rodriguez JJ, Sanchez PL, Marsal JR, Ribera A, Marrugat J, Bueno H, MASCARA study group. Patterns of use and effectiveness of early invasive strategy in non-ST-segment elevation acute coronary syndromes: an assessment by propensity score. Am Heart J. 2008 Nov;156(5):946-53. doi: 10.1016/j.ahj.2008.06.032
Reed SD, Anstrom KJ, Bakhai A, Briggs AH, Califf RM, Cohen DJ, Drummond MF, Glick HA, Gnanasakthy A, Hlatky MA, O'Brien BJ, Torti FMJr, Tsiatis AA, Wilan AR, Mark DB, Schulman KA. Conducting economic evaluations alongside multinational clinical trials: toward a research consensus. Am Heart J. 2005 Mar;149(3):434-43.