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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
Foraster M, Esnaola M, Lopez-Vicente M, Rivas I, Alvarez-Pedrerol M, Persavento C, Sebastian-Galles N, Pujol J, Dadvand P, Sunyer J. Exposure to road traffic noise and cognitive development in schoolchildren in Barcelona, Spain: a population-based cohort study. PLoS Med. 2022 Jun 2;19(6):e1004001. doi: 10.1371/journal.pmed.1004001
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
Sunyer J, Esnaola M, Alvarez-Pedrerol M, Forns J, Rivas I, Lopez-Vicente M, Suades-Gonzalez E, Foraster M, Garcia-Esteban R, Basagana X, Viana M, Cirach M, Moreno T, Alastuey A, Sebastian-Galles N, Nieuwenhuijsen M, Querol X. Association between traffic-related air pollution in schools and cognitive development in primary school children: a prospective cohort study. PLoS Med. 2015 Mar 3;12(3):e1001792. doi: 10.1371/journal.pmed.1001792