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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
Perez-Rodon J, Galve E, Perez-Bocanegra C, Soriano-Sanchez T, Recio-Iglesias J, Domingo-Baldrich E, Alzola-Guevara M, Ferriera-Gonzalez I, Marsal JR, Ribera-Sole A, Gutierrez Garcia-Moreno L, Cruz-Carlos LM, Rivas-Gandara N, Roca-Luque I, Francisco-Pascual J, Evangelista-Masip A, Moya-Mitjans A, Garcia-Dorado D. A risk score to predict the absence of left ventricular reverse remodeling: implications for the timing of ICD implantation in primary prevention. J Cardiol. 2018 May;71(5):505-12. doi: 10.1016/j.jjcc.2017.10.019
Epstein MM, Divine G, Chao CR, Wells KE, Feigelson HS, Scholes D, Roblin D, Yood MU, Engel LS, Taylor A, Fortuny J, Habel LA, Johnson CC. Statin use and risk of multiple myeloma: an analysis from the Cancer Research Network. Int J Cancer. 2017 Aug 1;141(3):480-7. doi: 10.1002/ijc.30745
Hoyo C, Schildkraut JM, Murphy SK, Chow WH, Vaughan TL, Risch H, Marks JR, Jirtle RL, Calingaert B, Mayne S, Fraumeni, Jr. J, Gammon MD. IGF2R polymorphisms and risk of esophageal and gastric adenocarcinomas. Int J Cancer. 2009 Dec 1;125(11):2673-8.
Maurel J, Nadal C, Garcia-Albeniz X, Gallego R, Carcereny E, Almendro V, Marmol M, Gallardo E, Maria Auge J, Longaron R, Martinez-Fernandez A, Molina R, Castells A, Gascon P. Serum matrix metalloproteinase 7 levels identifies poor prognosis advanced colorectal cancer patients. Int J Cancer. 2007 Sep 1;121(5):1066-71.
Fortuny J, Kogevinas M, Chang-Claude J, Gonzalez CA, Hours M, Jockel KH, Bolm-Audorff U, Lynge E, 't Mannetje A, Porru S, Ranft U, Serra C, Tzonou A, Wahrendorf J, Boffetta P. Tobacco, occupation and non-transitional-cell carcinoma of the bladder: an international case-control study. Int J Cancer. 1999 Jan 5;80(1):44-6. doi: 3.0.co;2-8">10.1002/(sici)1097-0215(19990105)80:1<44::aid-ijc9>3.0.co;2-8