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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
McSweeney L, Breckons M, Fattakhova G, Oluboyede Y, Vale L, Ternent L, Balp M-M, Doward L, Brass CA, Beyer F, Sanyal A, Anstee QM. Health-related quality of life and patient-reported outcome measures in NASH-related cirrhosis. JHEP Rep. 2020 Mar 6;2(3). doi: 10.1016/j.jhepr.2020.100099
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
Parekh D, Dancer RCA, Scott A, D'Souza VK, Howells PA, Mahida RY, Tang JCY, Cooper MS, Fraser WD, Tan L, Gao F, Martineau AR, Tucker O, Perkins GD, Thickett DR. Vitamin D to prevent lung injury following esophagectomy-a randomized, placebo-controlled trial. Crit Care Med. 2018 Dec;46(12):e1128-35. doi: 10.1097/CCM.0000000000003405
Parekh D, Patel JM, Scott A, Lax S, Dancer RC, D'Souza V, Greenwood H, Fraser WD, Gao F, Sapey E, Perkins GD, Thickett DR. Vitamin D deficiency in human and murine sepsis. Crit Care Med. 2017 Feb;45(2):282-9. doi: 10.1097/CCM.0000000000002095
Vaz LE, Kleinman KP, Kawai AT, Jin R, Kassler WJ, Grant PS, Rett MD, Goldmann DA, Calderwood MS, Soumerai SB, Lee GM. Impact of Medicare's Hospital-Acquired Condition policy on infections in safety net and non-safety net hospitals. Infect Control Hosp Epidemiol. 2015 Jun;36(6):649-55. doi: 10.1017/ice.2015.38.