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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
Cruz Rivera S, Aiyegbusi OL, Ives J, Draper H, Mercieca-Bebber R, Ells C, Hunn A, Scott JA, Fernandez CV, Dickens AP, Anderson N, Bhatnagar V, Bottomley A, Campbell L, Collett C, Collis P, Craig K, Davies H, Golub R, Gosden L, Gnanasakthy A, Haf Davies E, von Hildebrand M, Lord JM, Mahendraratnam N, Miyaji T, Morel T, Monteiro J, Zwisler AO, Peipert JD, Roydhouse J, Stover AM, Wilson R, Yap C, Calvert MJ. Ethical considerations for the inclusion of patient-reported outcomes in clinical research: the PRO ethics guidelines. JAMA. 2022 May 17;327(19):1910-9. doi: 10.1001/jama.2022.6421
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
Rodes-Cabau J, Horlick E, Ibrahim R, Cheema AN, Labinaz M, Nadeem N, Osten M, Cote M, Marsal JR, Rivest D, Marrero A, Houde C. Effect of clopidogrel and aspirin vs aspirin alone on migraine headaches after transcatheter atrial septal defect closure: the CANOA randomized clinical trial. JAMA. 2015 Nov 24;314(20):2147-54. doi: 10.1001/jama.2015.13919
Kroenke K, West SL, Swindle R, Gilsenan A, Eckert GJ, Dolor R, Stang P, Zhou XH, Hays R, Weinberger M. Similar effectiveness of paroxetine, fluoxetine, and sertraline in primary care: a randomized trial. JAMA. 2001 Dec 19;286(23):2947-55.