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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
Doward L, Twiss J, Breckons M, Balp MM, Pettersson B, McSweeney L, Oluboyede Y, Vale L, Brass C, Anstee QM, Sanyal AJ. Evaluation of the NASH-CHECK patient-reported outcome instrument for patients with nonalcoholic steatohepatitis (NASH) with compensated cirrhosis. Poster presented at the AASLD Liver Meeting; November 12, 2021. Anaheim, CA. [abstract] Hepatology. 2021 Oct 1; 74(S1):1007A. doi: 10.1002/hep.32188
Whalley D, Twiss J, Doward L, Balp MM, Brass C, Tietz A, Loeffler J, Lopez P, Lawitz EJ, Sanyal AJ. A novel patient-reported outcome measure indicates low burden of treatment among patients with non-alcoholic steatohepatitis: interim results from a phase 2 trial of tropifexor. Poster presented at the AASLD 2020 annual meeting; November 13, 2020. Boston, MA. [abstract] Hepatology. 2020 Oct 1; 72(S1):1005A. doi: 10.1002/hep.31579
Doward L, Twiss J, Whalley D, Balp MM, Brass C, Loeffler J, Cryer DR, Cai J, Lopez P, Lawitz EJ, Anstee QM, Sanyal AJ. Development of NASH-CHECK: a new patient-reported outcome measure for NASH. Poster presented at the AASLD 2020 annual meeting; November 13, 2020. Boston, MA. [abstract] Hepatology. 2020 Oct 1; 72(S1):1014A-5A. doi: 10.1002/hep.31579
Musara P, Milford C, Shapley-Quinn MK, Weinrib R, Mutero P, Odoom E, Mgodi NM, Chirenje ZM, Hanif H, Clark MR, Smit J, van der Straten A, Montgomery ET, Quatro Study Team. Preferences and acceptability of vaginal delivery forms for HIV prevention among women, male partners and key informants in South Africa and Zimbabwe: qualitative findings. AIDS Behav. 2020 Jun 25. doi: 10.1007/s10461-020-02949-4
Browne EN, Montgomery ET, Mansfield C, Boeri M, Mange B, Beksinska M, Schwartz JL, Clark MR, Doncel GF, Smit J, Chirenje ZM, van der Straten A. Efficacy is not everything: eliciting women's preferences for a vaginal HIV prevention product using a discrete-choice experiment. AIDS Behav. 2020 May;24(5):1443-51. doi: 10.1007/s10461-019-02715-1
Weinrib R, Browne EN, Shapley-Quinn MK, van der Straten A, Beksinska M, Mgodi N, Musara P, Mphili N, Schwartz JL, Ju S, Hanif H, Montgomery ET, Quatro Study Team. Perspectives from young South African and Zimbabwean women on attributes of four (placebo) vaginal microbicide delivery forms. AIDS Behav. 2020 Feb;24(2):637-47. doi: 10.1007/s10461-019-02576-8
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
Doward LC, Balp M-M, Twiss J, Slota C, Cryer D, Langford A, Collen R, Agashivala N, Brass CA, Anstee QM, Sanyal AJ. Understanding the patient-perceived impact of nonalcoholic steatohepatitis: raising the volume on a silent disease. Poster presented at the 2017 AASLD Liver Meeting; October 23, 2017. Washington, DC. [abstract] Hepatology. 2017 Jul; 66(1 Suppl):1182A.