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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
Omland LH, Holm-Hansen C, Lebech AM, Dessau RB, Bodilsen J, Andersen NS, Roed C, Christiansen CB, Ellermann-Eriksen S, Midgley S, Nielsen L, Benfield T, Hansen AE, Anderson CO, Rothman KJ, Sorensen HT, Fischer TK, Obel N. Long-term survival, health, social functioning, and education in patients with an enterovirus central nervous system infection, Denmark, 1997-2016. J Infect Dis. 2020 Jul 23;222(4):619-27. doi: 10.1093/infdis/jiaa151
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
Young-Xu Y, Van Aalst R, Mahmud SM, Rothman KJ, Thornton Snider J, Westreich D, Mor V, Gravenstein S, Lee JKH, Thommes EW, Decker MD, Chit A. Relative vaccine effectiveness of high-dose versus standard-dose influenza vaccines among Veterans Health Administration Patients. J Infect Dis. 2018 May;217(11):1718-27. doi: 10.1093/infdis/jiy088