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Publications
Melgar M, Abrams JY, Godfred-Cato S, Shah AB, Garg A, Strunk A, Narasimhan M, Koptyev J, Norden A, Musheyev D, Rashid F, Tannenbaum R, Estrada-Y-Martin RM, Patel B, Karanth S, Achenbach CJ, Hall GT, Hockney SM, Caputo M, Abbo LM, Beauchamps L, Morris SB, Cifuentes RO, de St Maurice A, Bell DS, Prabaker KK, Sanz Vidorreta FJ, Bryant E, Cohen DK, Mohan R, Libby CP, SooHoo S, Domingo TJ, Campbell AP, Belay ED. A multicenter retrospective cohort study to characterize patients hospitalized with MIS-A and COVID-19 in the United States, 2020-2021. Clin Infect Dis. 2023 Nov 17;77(10):1395-405. doi: 10.1093/cid/ciad374
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
MacDougall JE, Johnston JM, Lavins BJ, Nelson LM, Williams VS, Carson RT, Shiff SJ, Shi K, Kurtz CB, Baird MJ, Currie MG, Lembo AJ. An evaluation of the FDA Responder Endpoint for IBS-C clinical trials: analysis of data from linaclotide Phase 3 clinical trials. Neurogastroenterol Motil. 2013 Jun;25(6):481-6. doi: 10.1111/nmo.12089
Juliao PC, Maslanka S, Dykes J, Gaul L, Bagdure S, Granzow-Kibiger L, Salehi E, Zink D, Neligan RP, Barton-Behravesh C, Luquez C, Biggerstaff M, Lynch M, Olson C, Williams I, Barzilay EJ. National outbreak of type a foodborne botulism associated with a widely distributed commercially canned hot dog chili sauce. Clin Infect Dis. 2013 Feb;56(3):376-82. doi: 10.1093/cid/cis901
Sheth AN, Wiersma P, Atrubin D, Dubey V, Zink D, Skinner G, Doerr F, Juliao P, Gonzalez G, Burnett C, Drenzek C, Shuler C, Austin J, Ellis A, Maslanka S, Sobel J. International outbreak of severe botulism with prolonged toxemia caused by commercial carrot juice. Clin Infect Dis. 2008 Nov 15;47(10):1245-51. doi: 10.1086/592574.