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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
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