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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
Kapral MK, Austin PC, Jeyakumar G, Hall R, Chu A, Khan AM, Jin AY, Martin C, Manuel D, Silver FL, Swartz RH, Tu JV. Rural-urban differences in stroke risk factors, incidence, and mortality in people with and without prior stroke. Circ Cardiovasc Qual Outcomes. 2019 Feb;12(2):e004973. doi: 10.1161/CIRCOUTCOMES.118.004973
Ferreira-Gonzalez I, Marsal JR, Mitjavila F, Parada A, Ribera A, Cascant P, Soriano N, Sanchez PL, Aros F, Heras M, Bueno H, Marrugat J, Cunat J, Civeira E, Permanyer-Miralda G. Patient registries of acute coronary syndrome: assessing or biasing the clinical real world data? Circ Cardiovasc Qual Outcomes. 2009 Nov;2(6):540-7. doi: 10.1161/CIRCOUTCOMES.108.844399