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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
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
Mercieca-Bebber R, Rouette J, Calvert M, King MT, McLeod L , Holch P, Palmer MJ, Brundage M, ISOQOL Best Practice for PROs-Reporting Task Force. Preliminary evidence on the uptake, use, and benefits of the CONSORT-PRO extension . Qual Life Res. 2017 Jun;26(6):1427-37. doi: 10.1007/s11136-017-1508-6
Luquiens A, Whalley D , Crawford SR , Laramée P, Doward L , Price M , Hawken N, Dorey J, Owens L, Llorca PM, Falissard B, Aubin HJ. Development of the Alcohol Quality of Life Scale (AQoLS): a new patient-reported outcome measure to assess health-related quality of life in alcohol use disorder . Qual Life Res. 2015 Jun;24(6):1471-81. doi: 10.1007/s11136-014-0865-7
Reeve BB, Wyrwich KW, Wu AW, Velikova G, Terwee CB, Snyder CF, Schwartz C, Revicki DA, Moinpour CM, McLeod LD , Lyons JC, Lenderking WR, Hinds PS, Hays RD, Greenhalgh J, Gershon R, Feeny D, Fayers PM, Cella D, Brundage M, Ahmed S, Aaronson NK, Butt Z. ISOQOL recommends minimum standards for patient-reported outcome measures used in patient-centered outcomes and comparative effectiveness research . Qual Life Res. 2013 Oct;22(8):1889-905. doi: 10.1007/s11136-012-0344-y
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.