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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
Janssens R, Lang T, Vallejo A, Galinsky J , Plate A, Morgan K, Cabezudo E, Silvennoinen R, Coriu D, Badelita S, Irimia R, Anttonen M, Manninen RL, Schoefs E, Vandebroek M, Vanhellemont A, Delforge M, Stevens H, Simoens S, Huys I. Patient preferences for multiple myeloma treatments: a multinational qualitative study . Front Med (Lausanne). 2021 Jul 6;8:686165. doi: 10.3389/fmed.2021.686165
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.