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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
Soler MJ, Ribera A, Marsal JR, Mendez AB, Andres M, Azancot MA, Oristrell G, Mendez-Boo L, Cohen J, Barrabes JA, Ferreira-Gonzalez I, Vall d’Hebron COVID-19 Working Group. Association of renin-angiotensin system blockers with COVID-19 diagnosis and prognosis in patients with hypertension: a population-based study. Clin Kidney J. 2021 Sep 3;15(1):79-94. doi: 10.1093/ckj/sfab161
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
Gilbertson DT, Rothman KJ, Chertow GM, Bradbury BD, Brookhart MA, Liu L, Winkelmayer WC, Sturmer T, Monda KL, Herzog CA, Ashfaq A, Collins AJ, Wetmore JB. Excess deaths attributable to influenza-like illness in the ESRD population. J Am Soc Nephrol. 2019 Feb;30(2):346-53. doi: 10.1681/ASN.2018060581
Chertow GM, Liu J, Monda KL, Gilbertson DT, Brookhart MA, Beaubrun AC, Winkelmayer WC, Pollock A, Herzog CA, Ashfaq A, Sturmer T, Rothman KJ, Bradbury BD, Collins AJ. Epoetin alfa and outcomes in dialysis amid regulatory and payment reform. J Am Soc Nephrol. 2016 Oct;27(10):3129-38. doi: 10.1681/ASN.2015111232