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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
Walley D, Twiss J, Doward L, Balp MM, Brass C, Loeffler J, Lopez P, Cai J, Cryer D, Anstee Q, Sanyal A. Patient-reported symptoms and impact in nonalcoholic steatohepatitis: an evaluation of the NASH-CHECK, a novel PROM in NASH. Poster presented at the EASL: The Digital International Liver Congress; August 27, 2020. Digital. [abstract] J Hepatol. 2020 Jan; 73:S422-3. doi: 10.1016/S0168-8278(20)33053-1
Breckons M, Doward L, McSweeney L, Balp MM, Twiss J, Oluboyede Y, Vale L, Ternent L, Brass C, Sanyal A, Anstee Q. Evaluating the patient-perceived impact of non-alcoholic steatohepatitis with compensated cirrhosis. Poster presented at the EASL: The Digital International Liver Congress; August 27, 2020. digital. [abstract] J Hepatol. 2020 Jan; 73:S423. doi: 10.1016/S0168-8278(20)33053-1
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 L, Balp MM, Twiss J, Slota C, Cryer D, Langford A, Collen R, Agashivala N, Brass C, Anstee QM, Sanyal A. Measuring what matters to patients: the development of the NASH-CHECK, a new patient-reported outcome instrument for nonalcoholic steatohepatitis. Poster presented at the 2018 EASL International Liver Conference; April 13, 2018. Paris, France. [abstract] J Hepatol. 2018 Apr; 68(Suppl 1):S570.
Doward LC, Balp MM, Stewart KE, Cryer D, Langford A, Twiss J, Agashivala N, Brass C, Anstee QM, Sanyal A. Exploring the patient perceived impact of non-alcoholic steatohepatitis. Poster presented at the 2017 EASL International Liver Conference; April 2017. Amsterdam, The Netherlands. [abstract] J Hepatol. 2017 Apr; 66(1 Supplement):S422-3. doi: 10.1016/S0168-8278(17)31208-4
Williams AG, Dhamrait SS, Wootton PT, Day SH, Hawe E, Payne JR, Myerson SG, World M, Budgett R, Humphries SE, Montgomery HE. Bradykinin receptor gene variant and human physical performance. J Appl Physiol. 2004;96(3):938-42.