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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
Shah R, Girard N, Nagar SP, Griesinger F, Roeper J, Davis KL, Karimi P, Sawyer W, Yu N, Taylor A, Feliciano J. European and US real-world treatment patterns in patients with epidermal growth factor receptor mutation-positive non-small cell lung cancer: a retrospective medical record review. Drugs Real World Outcomes. 2021 Dec;8(4):537-45. doi: 10.1007/s40801-021-00261-8
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
Kuiper JG, van Herk-Sukel MPP, Castellsague J, Pottegard A, Berglind IA, Dedman D, Gutierrez L, Calingaert B, Hallas J, Sundstrom A, Gallagher AM, Kaye JA, Pardo C, Rothman KJ, Perez-Gutthann S. Use of topical tacrolimus and topical pimecrolimus in four European countries: a multicentre database cohort study. Drugs Real World Outcomes. 2018 Jun;5(2):109-16. doi: 10.1007/s40801-018-0133-1