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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
Pascal R, Crovetto F, Casas I, Youssef L, Trilla C, Larroya M, Cahuana A, Boada D, Foraster M, Llurba E, Sunyer J, Crispi F, Gratacos E, Gomez-Roig MD. Impact of the COVID-19 pandemic on maternal well-being during pregnancy. J Clin Med. 2022 Apr 15;11(8):2212. doi: 10.3390/jcm11082212
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
Vilanova MB, Franch-Nadal J, Falguera M, Marsal JR, Canivell S, Rubinat E, Miro N, Mollo A, Mata-Cases M, Gratacos M, Castelblanco E, Mauricio D. Prediabetes is independently associated with subclinical carotid atherosclerosis: an observational study in a non-urban Mediterranean population. J Clin Med. 2020 Jul 7;9(7):2139. doi: 10.3390/jcm9072139
Koziel M, Mazurek M, Teutsch C, Diener HC, Dubner SJ, Halperin JL, Ma CS, Rothman KJ, Brandes A, Paquette M, Zint K, Franca LR, Lu S, Bartels DB, Huisman MV, Lip GYH. Persistence with anticoagulation for atrial fibrillation: report from the GLORIA-AF Phase III 1-year follow-up. J Clin Med. 2020 Jun 23;9(6):1969. doi: 10.3390/jcm9061969
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