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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
Montazeri P, Fossati S, Clemente DBP, Cirugeda L, Elosua R, Fernandez-Barres S, Fochs S, Garcia-Esteban R, Marquez S, Pey N, Nawrot TS, Vrijheid M. Early-childhood BMI trajectories in relation to preclinical cardiovascular measurements in adolescence. J Dev Orig Hlth Dis. 2022 Jun;13(3):322-9. doi: 10.1017/S2040174421000441
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
Polanska K, Kaluzny P, Aubert AM, Bernard JY, Duijts L, El Marroun H, Hanke W, Hebert JR, Heude B, Jankowska A, Mancano G, Mensink-Bout SM, Relton C, Shivappa N, Suderman M, Trafalska E, Wesolowska E, Garcia-Esteban R, Guxens M, Casas M, Phillips CM. Dietary quality and dietary inflammatory potential during pregnancy and offspring emotional and behavioral symptoms in childhood: an individual participant data meta-analysis of four European cohorts. Biol Psychiatry. 2021 Mar 15;89(6):550-9. doi: 10.1016/j.biopsych.2020.10.008
Phiri K, Hallas J, Linder M, Margulia A, Suehs B, Arana A, Bahmanyar S, Enger C, Horter L, Odsbu I, Olesen M, Perez-Gutthann S, Sahlertz Kristiansen N, Appenteng K, de Vogel S, Seeger J. A study of cancer occurrence in users of mirabegron and antimuscarinic treatments for overactive bladder. Poster presented at the American Urological Association 2020 Conference (Conference cancelled); May 2020. Washington, DC. [abstract] J Urol. 2020 Apr; 203(Supplement 4):e910-1. doi: 10.1097/JU.0000000000000931.010
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