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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
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
Stempel DA, Szefler SJ, Pedersen S, Zeiger RS, Yeakey AM, Lee LA, Liu AH, Mitchell H, Kral KM, Raphiou IH, Prillaman BA, Buaron SK, Kirby SY, Pascoe SJ. Safety of adding salmeterol to fluticasone propionate in children with asthma. N Engl J Med. 2016 Sep 1;375(9):840-9. doi: 10.1056/NEJMoa1606356
Lee GM, Kleinman K, Soumerai SB, Tse (Kawai) A, Cole D, Fridkin SK, Horan T, Platt R, Gay C, Kassler W, Goldmann DA, Jernigan J, Jha AK. Effect of nonpayment for preventable infections in U.S. hospitals. N Engl J Med. 2012 Oct 11;367(15):1428-37. doi: 10.1056/NEJMsa1202419
Khalil S, Rodarte A, Weldon BC, Weinstein M, Grunwald Z, Ginsberg B, Kaye R, Otto A, Wheeler M, Lawhorn CD, Prillaman BA, Creed M. Intravenous ondansetron in established postoperative emesis in children. S3A-381 study group. Anesthesiology. 1996 Aug;85(2):270-6. doi: 10.1097/00000542-199608000-00007