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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
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
Hoffman E, Mladsi DM, Cryer B, Hopkins W, Brater DC, Parikh R, Goyal R, Castellsague J, Stafkey-Mailey D, Young C. Dose-related risks of cardiovascular, gastrointestinal, and renal adverse events associated with meloxicam among patients with osteoarthritis: an observational study using US claims data. Poster presented at the 2016 ACR/ARHP Annual Meeting; November 15, 2016. Washington, DC. [abstract] Arthritis Rheumatol. 2016 Sep 28; 68(Suppl 10).
Gossec L, Kvien TK, Conaghan PG, Ostergaard M, Canete JD, Gaillez C, Mpofu S, Sherif B, Jugl S. Relationship between improvements in fatigue and signs & symptoms of active psoriatic: arthritis a sub-analysis of a phase 3 study with secukinumab. Poster presented at the 2015 ACR/ARHP Annual Meeting; November 10, 2015. San Francisco, CA. [abstract] Arthritis Rheumatol. 2015 Sep 29; 67(Suppl 10).
Helliwell PS, Doward L, Whalley D, Tennant A, McKenna S, Reynolds S, Kay L, Spoorenberg A, van der Heijde Leeds D. Psychometric and scaling properties of a new quality of life instrument specific to ankylosing spondylitis (AS). Poster presented at the 1999 ACR/ARHP Annual Scientific Meeting; November 14, 1999. Boston, MA. [abstract] Arthritis Rheumatol. 1999 Sep; 42(S9):s72.