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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
Soler MJ, Ribera A, Marsal JR , Mendez AB, Andres M, Azancot MA, Oristrell G, Mendez-Boo L, Cohen J, Barrabes JA, Ferreira-Gonzalez I, Vall d’Hebron COVID-19 Working Group. Association of renin-angiotensin system blockers with COVID-19 diagnosis and prognosis in patients with hypertension: a population-based study . Clin Kidney J. 2021 Sep 3;15(1):79-94. doi: 10.1093/ckj/sfab161
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
Francois C, Danchenko N, Williams VS , Nelson LM , Williams NJ, Yarr SR , DiBenedetti DB , Lancon C. Psychometric evaluation of the depression and family functioning scale (DFFS) . Poster presented at the Second Meeting on Patient Reported Outcomes and Person Centered Care in Mental Health – Diagnosis, Prevention and Treatment in Mental Health: Effectiveness and Comparative Effectiveness; September 26, 2014. Washington, DC. [abstract] J Ment Health Policy Econ. 2014 Sep; 17(Suppl 1):S5-6.