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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
Juliao P, Wurst K, Pimenta JM, Gemzoe K, Landy H, Moody MA, Tilson H, Covington D, Moore T, Marino R, Gilbride J, Liu A, Meizlik P, Petri M. Belimumab use during pregnancy: interim results of the belimumab pregnancy registry. Birth Defects Res Part A Clin Mol Teratol. 2023 Jan 15;115(2):188-204. doi: 10.1002/bdr2.2091
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
Polanska K, Kaluzny P, Aubert AM, Bernard JY, Duijts L, El Marroun H, Hanke W, Hebert JR, Heude B, Jankowska A, Mancano G, Mensink-Bout SM, Relton C, Shivappa N, Suderman M, Trafalska E, Wesolowska E, Garcia-Esteban R, Guxens M, Casas M, Phillips CM. Dietary quality and dietary inflammatory potential during pregnancy and offspring emotional and behavioral symptoms in childhood: an individual participant data meta-analysis of four European cohorts. Biol Psychiatry. 2021 Mar 15;89(6):550-9. doi: 10.1016/j.biopsych.2020.10.008
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
Tennis P, Chan KA, Curkendall SN, Li DK, Mines D, Peterson C, Andrews EB, Calingaert B, Chen HY, Deshpande G, Everage N, Holick CN, Meyer NM, Nkhoma ET, Quinn S, Rothman KJ, Esposito DB. Topiramate use during pregnancy and major congenital malformations in multiple populations. Birth Defects Res Part A Clin Mol Teratol. 2015 Apr;103(4):269-75. doi: 10.1002/bdra.23357.