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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
Rebordosa C, Plana E, Rubino A, Aguado J, Martinez D, Lei A, Daoud S, Saigi-Morgui N, Perez-Gutthann S, Rivero-Ferrer E. Risk assessment of acute myocardial infarction and stroke associated with long-acting muscarinic antagonists, alone or in combination, versus long-acting beta2-agonists. Int J Chron Obstruct Pulmon Dis. 2022 Aug 2;17:1715-33. doi: 10.2147/COPD.S363997
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
Naranjo L, Torres-Duque CA, Colodenco D, Lopardo G, Rodriguez P, Agra de Albuquerque-Neto A, Hernandez-Zenteno RJ, Septien L, Chandler R, Ortega-Barria E, Juliao P, Guzman-Holst A. Highlights of an expert advisory board on Acute Exacerbations of Chronic Obstructive Pulmonary Disease (AE-COPD) in Latin America. Int J Chron Obstruct Pulmon Dis. 2020 Aug 6;15:1919-29. doi: 10.2147/COPD.S261258
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
Price KJ, Knowles JA, Fox M, Wales JK, Heller S, Eiser C, Freeman JV, Brennan A, Basarir H, Stevens K, McPherson A. Effectiveness of the Kids in Control of Food (KICk–OFF) structured education course for 11–16 year olds with type 1 diabetes. Diabet Med. 2016 Feb;33(2):192-203. doi: 10.1111/dme.12881
Goatman KA, Philip S, Fleming AD, Harvey RD, Swa KK, Styles C, Black M, Sell G, Lee N, Sharp PF, Olson JA. External quality assurance for image grading in the Scottish diabetic retinopathy screening programme. Diabet Med. 2012 Jun 1;29(6):776-83. doi: 10.1111/j.1464-5491.2011.03504.x