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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
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
Chamandi C, Barbanti M, Munoz-Garcia A, Latib A, Nombela-Franco L, Gutierrez-Ibanez E, Veiga-Fernandez G, Cheema AN, Cruz-Gonzalez I, Serra V, Tamburino C, Mangieri A, Colombo A, Jimenez-Quevedo P, Elizaga J, Laughlin G, Lee DH, Garcia Del Blanco B, Rodriguez-Gabella T, Marsal JR , Cote M, Philippon F, Rodes-Cabau J. Long-term outcomes in patients with new permanent pacemaker implantation following transcatheter aortic valve replacement . JACC Cardiovasc Interv. 2018 Feb 12;11(3):301-10. doi: 10.1016/j.jcin.2017.10.032
Abdul-Jawad Altisent O, Durand E, Munoz-Garcia AJ, Nombela-Franco L, Cheema A, Kefer J, Gutierrez E, Benitez LM, Amat-Santos IJ, Serra V, Eltchaninoff H, Alnasser SM, Elizaga J, Dager A, Garcia Del Blanco B, Ortas-Nadal MDR, Marsal JR , Campelo-Parada F, Regueiro A, Del Trigo M, Dumont E, Puri R, Rodes-Cabau J. Reply: antithrombotic regimen in post-TAVR atrial fibrillation: not an easy decision . JACC Cardiovasc Interv. 2016 Nov 28;9(22):2366-8. doi: 10.1016/j.jcin.2016.09.038
Abdul-Jawad Altisent O, Durand E, Munoz-Garcia AJ, Nombela-Franco L, Cheema A, Kefer J, Gutierrez E, Benitez LM, Amat-Santos IJ, Serra V, Eltchaninoff H, Alnasser SM, Elizaga J, Dager A, Garcia Del Blanco B, Ortas-Nadal Mdel R, Marsal JR , Campelo-Parada F, Regueiro A, Del Trigo M, Dumont E, Puri R, Rodes-Cabau J. Warfarin and antiplatelet therapy versus Warfarin alone for treating patients with atrial fibrillation undergoing transcatheter aortic valve replacement . JACC Cardiovasc Interv. 2016 Aug 22;9(16):1706-17. doi: 10.1016/j.jcin.2016.06.025
Boccia S, Rothman KJ , Panic N, Flacco ME, Rosso A, Pastorino R, Manzoli L, La Vecchia C, Villari P, Boffetta P, Ricciardi W, Ioannidis JP. Registration practices for observational studies on ClinicalTrials.gov indicated low adherence . J Clin Epidemiol. 2016 Feb;70:176-82. doi: 10.1016/j.jclinepi.2015.09.009