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Publications
Bhashyam SS, Shane LG, Lewis HB, de la Cruz M, Galinsky J, Demchuk K, Waite NM, Lazarus JV, Salisbury DM. Results from the COVID-19 vaccines discrete choice experiment pre-test qualitative interviews in Canada, Germany, the UK, and US general population. Poster presented at the ID Week 2023; October 3, 2023. Washington, DC. [abstract] Open Forum Infect Dis. 2023 Nov 27; 10(Suppl 2):S263-4. doi: 10.1093/ofid/ofad500.1208
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
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
Nance R, Vannappagari V, Johannes C, Calingaert B, Saltus C, Boswell S, Rodriguez B, Moore R, Eron J, Geng E, Mathews WC, Saag MS, Kitahata M, Delaney JAC, Crane HM, Centers For AIDS Research Network of Integrated Clinical Systems (CNICS). Viral failure among persons living with HIV initiating dolutegravir-based vs. other recommended regimens in real-world clinical care settings. Poster presented at the IDWeek 2017 Conference; October 2017. San Diego, CA. [abstract] Open Forum Infect Dis. 2017 Oct 4; 4((Suppl 1)):S39. doi: 10.1093/ofid/ofx162.095
Baker M, Jankosky C, Yih K, Gruber S, Li L, Cocoros N, Lipowicz H, Coronel-Moreno C, Feibelmann S, Lin N, McMahill-Walraven C, Menschik D, Selvan M, Selvam N, Tilney RC, Zichittella L, Lee G, Kawai AT. The risk of febrile seizures following influenza and 13-valent pneumococcal conjugate vaccines. Poster presented at the 2017 Infectious Disease Week Conference; October 6, 2017. San Diego, CA. [abstract] Open Forum Infect Dis. 2017 Oct; 4(Suppl 1):S464-5. doi: 10.1093/ofid/ofx163.1187
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.