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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
Koziel M, Teutsch C, Bayer V, Lu S, Gurusamy VK, Halperin JL, Rothman KJ , Diener HC, Ma CS, Huisman MV, Lip GYH, GLORIA‐AF Investigators. Changes in anticoagulant prescription patterns over time for patients with atrial fibrillation around the world . J Arrhythm. 2021 Jul 10;37(4):990-1006. doi: 10.1002/joa3.12588
Ma C, Riou Franca L, Lu S, Diener H-C, Dubner SJ, Halperin JL, Li Q, Paquette M, Teutsch C, Huisman MV, Lip GYH, Rothman KJ . Stroke prevention in atrial fibrillation changes after dabigatran availability in China: the GLORIA‐AF registry . J Arrhythm. 2020 Mar 10;36(3):408-16. doi: 10.1002/joa3.12321
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
Allhusen V, Belsky J, Kersey HB, Booth C, Bradley R, Brownell CA, Burchinal M, Caldwell B, Campbell SB, Clarke-Stewart KA, Cox M, Friedman SL, Hirsh-Pasek K, Houts R, Huston A, Jaeger E, Kelly JF, Knoke B, Kochanoff A, Marshall N, McCartney K, McLeod L , Morrison FJ, O'Brien M, Owen MT, Payne C, Phillips D, Pianta R, Robeson WW, Spieker S, Vandell DL, Wallner-Allen KE. Do children's attention processes mediate the link between family predictors and school readiness? Dev Psychol. 2003 Jan 1;39(3):581-93.