Publications

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Displaying 1 through 15 of 15 publications.

Yee KS, Alexanderian D, Martin S, Olayinka-Amao B, Whiteman DAH. Clinical investigator perspectives on patient outcomes in children with neuronopathic mucopolysaccharidosis II during intrathecal idursulfase-IT treatment. Orphanet J Rare Dis. 2024 Apr 12;19(1):148. doi: 10.1186/s13023-024-03147-4

Hu M, Shoaibi A, Feng Y, Lloyd PC, Wong HL, Smith ER, Amend KL, Kline A, Beachler DC, Gruber JF, Mitra M, Seeger JD, Harris C, Secora A, Obidi J, Wang J, Song J, McMahill-Walraven CN, Reich C, McEvoy R, Do R, Chillarige Y, Clifford R, Cooper DD, Forshee RA, Anderson SA. Safety of ancestral monovalent BNT162b2, mRNA-1273, and NVX-CoV2373 COVID-19 vaccines in US children aged 6 months to 17 years. JAMA Netw Open. 2024 Apr 1;7(4):e248192. doi: 10.1001/jamanetworkopen.2024.8192

Yee KS, Lewis S, Evans E, Romano (DeMuro) C, Alexanderian D. Caregiver experiences and observations of intrathecal idursulfase-IT treatment in a phase 2/3 trial in pediatric patients with neuronopathic mucopolysaccharidosis II. Orphanet J Rare Dis. 2024 Mar 10;19(1):100. doi: 10.1186/s13023-024-03034-y

Yee KS, Chirila C, Davenport E, Mladsi D, Barnett C, Kronenberger WG. A post hoc analysis of Projected Retained Ability Scores (PRAS) for the longitudinal assessment of cognitive functioning in patients with neuronopathic mucopolysaccharidosis II receiving intrathecal idursulfase-IT. Orphanet J Rare Dis. 2023 Nov 2;18(1):343. doi: 10.1186/s13023-023-02957-2

Hansford HJ, Cashin AG, Jones MD, Swanson SA, Islam N, Douglas SRG, Rizzo RRN, Devonshire JJ, Williams SA, Dahabreh IJ, Dickerman BA, Egger M, Garcia-Albeniz X, Golub RM, Lodi S, Moreno-Betancur M, Pearson SA, Schneeweiss S, Sterne JAC, Sharp MK, Stuart EA, Hernan MA, Lee H, McAuley JH. Reporting of observational studies explicitly aiming to emulate randomized trials: a systematic review. JAMA Netw Open. 2023 Sep 5;6(9):e2336023. doi: 10.1001/jamanetworkopen.2023.36023

Schneider KL, Bell EJ, Zhou CK, Yang G, Lloyd P, Clarke TC, Wilkinson M, Myers EE, Amend KL, Seeger JD, Chillarige Y, Forshee RA, Shoaibi A, Anderson SA, Wong HL. Use of immunization information systems in ascertainment of COVID-19 vaccinations for claims-based vaccine safety and effectiveness studies. JAMA Netw Open. 2023 May 1;6(5):e2313512. doi: 10.1001/jamanetworkopen.2023.13512.

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

Armstrong MA, Raine-Bennett T, Reed SD, Gatz J, Getahun D, Schoendorf J, Postlethwaite D, Fassett MJ, Peipert JF, Saltus CW, Merchant M, Alabaster A, Zhou X, Ichikawa L, Shi JM, Chiu VY, Xie F, Hunter S, Wang J, Ritchey ME, Chillemi G, Im TM, Takhar HS, Pisa F, Asiimwe A, Anthony MS. Association of the timing of postpartum intrauterine device insertion and breastfeeding with risks of intrauterine device expulsion. JAMA Netw Open. 2022 Feb 28;5(2):e2148474. doi: 10.1001/jamanetworkopen.2021.48474

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

Foreman PK, Margulis AV, Alexander K, Shediac R, Calingaert B, Harding A, Pladevall-Vila M, Landis S. Birth prevalence of phenylalanine hydroxylase deficiency: a systematic literature review and meta-analysis. Orphanet J Rare Dis. 2021 Jun 3;16(1):253. doi: 10.1186/s13023-021-01874-6

Petito LC, Garcia-Albeniz X, Logan RW, Howlader N, Mariotto AB, Dahabreh IJ, Hernan MA. Estimates of overall survival in patients with cancer receiving different treatment regimens: emulating hypothetical target trials in the Surveillance, Epidemiology, and End Results (SEER)–Medicare linked database. JAMA Netw Open. 2020 Mar 2;3(3):e200452. doi: 10.1001/jamanetworkopen.2020.0452

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

Harrington M, Whalley D, Twiss J, Rushton R, Martin S, Huynh L, Yang H. Insights into the natural history of metachromatic leukodystrophy from interviews with caregivers. Orphanet J Rare Dis. 2019 Apr 29;14(1):89. doi: 10.1186/s13023-019-1060-2

Wheeler AC, Sacco P, Cabo R. Unmet clinical needs and burden in Angelman syndrome: a review of the literature. Orphanet J Rare Dis. 2017 Oct 16;12(1):164. doi: 10.1186/s13023-017-0716-z

Herring WL, Herrmann JW. The single-day surgery scheduling problem: sequential decision-making and threshold-based heuristics. OR spectrum: quantitative approaches in management. 2012 Apr 1;34(2):429-59.