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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
Sukumaran L, McCarthy NL, Kharbanda EO, Vazquez-Benitez G, Lipkind HS, Jackson L, Klein NP, Naleway AL, McClure DL, Hechter RC, Kawai AT, Glanz JM, Weintraub ES. Infant hospitalizations and mortality after maternal vaccination. Pediatrics. 2018 Mar;141(3):e20173310. doi: 10.1542/peds.2017-3310
Vandenberghe F, Gholam-Rezaee M, Saigi-Morgui N, Delacretaz A, Choong E, Solida-Tozzi A, Kolly S, Thonney J, Gallo SF, Hedjal A, Ambresin AE, von Gunten A, Conus P, Eap CB. Importance of early weight changes to predict long-term weight gain during psychotropic drug treatment. J Clin Psychiatry. 2015 Nov;76(11):e1417-23. doi: 10.4088/JCP.14m09358.
Guxens M, Mendez MA, Molto-Puigmarti C, Julvez J, Garcia-Esteban R, Forns J, Ferrer M, Vrijheid M, Lopez-Sabater MC, Sunyer J. Breastfeeding, long-chain polyunsaturated fatty acids in colostrum, and infant mental development. Pediatrics. 2011 Oct;128(4):e880-9. doi: 10.1542/peds.2010-1633.