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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
Hanna MG, Badrising UA, Benveniste O, Lloyd TE, Needham M, Chinoy H, Aoki M, Machado PM, Liang C, Reardon KA, de Visser M, Ascherman DP, Barohn RJ, Dimachkie MM, Miller JAL, Kissel JT, Joyce NC, Van den Bergh P, Baets J, De Bleecker JL, Karam C, David WS, Mirabella M, Nations SP, Jung HH, Pegoraro E, Maggi L, Rodolico C, Filosto M, Shaibani AL, Sivakumar K, Goyal NA, Mori-Yoshimura M, Yamashita S, Suzuki N, Katsuno M, Murata K, Nodera H, Nishino I, Romano C , Williams VSL , Vissing J, Zhang Auberson L, Wu M, de Vera A, Papanicolaou DA, Amato AA, RESILIENT Study Group. Safety and efficacy of intravenous bimagrumab in inclusion body myositis: a phase 2b, randomised, double-blind, placebo-controlled study (RESILIENT) . Lancet Neurol. 2019 Sep;18(9):834-44.
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
Montazeri P, Vrijheid M, Martinez D , Basterrechea M, Fernandez-Somoano A, Guxens M, Iniguez C, Lertxundi A, Murcia M, Tardon A, Sunyer J, Valvi D. Maternal metabolic health parameters during pregnancy in relation to early childhood BMI trajectories . Obesity (Silver Spring). 2018 Mar;26(3):588-96. doi: 10.1002/oby.22095
Robinson O, Martinez D , Aurrekoetxea JJ, Estarlich M, Somoano AF, Iniguez C, Santa-Marina L, Tardon A, Torrent M, Sunyer J, Valvi D, Vrijheid M. The association between passive and active tobacco smoke exposure and child weight status among Spanish children . Obesity (Silver Spring). 2016 Aug;24(8):1767-77. doi: 10.1002/oby.21558
Valvi D, Mendez MA, Garcia-Esteban R , Ballester F, Ibarluzea J, Goni F, Grimalt JO, Llop S, Marina LS, Vizcaino E, Sunyer J, Vrijheid M. Prenatal exposure to persistent organic pollutants and rapid weight gain and overweight in infancy . Obesity (Silver Spring). 2014 Feb;22(2):488-96. doi: 10.1002/oby.20603