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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
Casas G, Limeres J, Oristrell G, Gutierrez-Garcia L, Andreini D, Borregan M, Larrañaga-Moreira JM, Lopez-Sainz A, Codina-Solà M, Teixido-Tura G, Sorolla-Romero JA, Fernández-Álvarez P, González-Carrillo J, Guala A, La Mura L, Soler-Fernández R, Sao Avilés A, Santos-Mateo JJ, Marsal JR, Ribera A, de la Pompa JL, Villacorta E, Jiménez-Jáimez J, Ripoll-Vera T, Bayes-Genis A, Garcia-Pinilla JM, Palomino-Doza J, Tiron C, Pontone G, Bogaert J, Aquaro GD, Gimeno-Blanes JR, Zorio E, Garcia-Pavia P, Barriales-Villa R, Evangelista A, Masci PG, Ferreira-González I, Rodríguez-Palomares JF. Clinical risk prediction in patients with left ventricular myocardial noncompaction. J Am Coll Cardiol. 2021 Aug 17;78(7):643-62. doi: 10.1016/j.jacc.2021.06.016
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
Paquette M, Riou Franca L, Teutsch C, Diener HC, Lu S, Dubner SJ, Ma CS, Rothman KJ, Zint K, Halperin JL, Huisman MV, Lip GYH, Nieuwlaat R. Persistence with dabigatran therapy at 2 years in patients with atrial fibrillation. J Am Coll Cardiol. 2017 Sep 26;70(13):1573-83. doi: 10.1016/j.jacc.2017.07.793
Huisman MV, Rothman KJ, Paquette M, Teutsch C, Diener HC, Dubner SJ, Halperin JL, Ma CS, Zint K, Elsaesser A, Bartels DB, Lip GY, GLORIA-AF Investigators. The changing landscape for stroke prevention in AF: findings from the GLORIA-AF Registry Phase 2. J Am Coll Cardiol. 2017 Feb 21;69(7):777-85.
Ferreira-Gonzalez I, Marsal JR, Ribera A, Permanyer-Miralda G, Garcia-Del Blanco B, Marti G, Cascant P, Masotti-Centol M, Carrillo X, Mauri J, Batalla N, Larrousse E, Martin E, Serra A, Rumoroso JR, Ruiz-Salmeron R, De La Torre JM, Cequier A, Gomez-Hospital JA, Alfonso F, Martin-Yuste V, Sabate M, Garcia-Dorado D. Double antiplatelet therapy after drug-eluting stent implantation: risk associated with discontinuation within the first year. J Am Coll Cardiol. 2012 Oct 9;60(15):1333-9. doi: 10.1016/j.jacc.2012.04.057