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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
Rubio-Casadevall J, Martinez-Trufero J, Garcia-Albeniz X, Calabuig S, Lopez-Pousa A, Del Muro JG, Fra J, Redondo A, Lainez N, Poveda A, Valverde C, De Juan A, Sevilla I, Casado A, Andres R, Cruz J, Martin-Broto J, Maurel J, Spanish Group for Research on Sarcoma. Role of surgery in patients with recurrent, metastatic, or unresectable locally advanced gastrointestinal stromal tumors sensitive to imatinib: a retrospective analysis of the Spanish Group for Research on Sarcoma (GEIS). Ann Surg Oncol. 2015 Sep;22(9):2948-57. doi: 10.1245/s10434-014-4360-8
Forns J, Vegas O, Julvez J, Garcia-Esteban R, Rivera M, Lertxundi N, Guxens M, Fano E, Ferrer M, Grellier J, Ibarluzea J, Sunyer J. Association between child cortisol levels in saliva and neuropsychological development during the second year of life. Stress Health. 2014 Apr;30(2):142-8. doi: 10.1002/smi.2504
Martinez-Fernandez A, Garcia-Albeniz X, Pineda E, Visa L, Gallego R, Codony-Servat J, Auge JM, Longaron R, Gascon P, Lacy A, Castells A, Maurel J. Serum matrilysin levels predict outcome in curatively resected colorectal cancer patients. Ann Surg Oncol. 2009 May;16(5):1412-20. doi: 10.1245/s10434-009-0405-9