Publications

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Displaying 1 through 15 of 15 publications.

Grivas P, Barata P, Moon H, Gupta S, Hutson T, Sternberg CN, Brown JR, Dave V, Downey C, Shillington AC, Katzenstein HM, Kirker M, Hanson S, Liu FX, Morris V, Bhanegaonkar A, Sonpavde GP. Avelumab first-line maintenance for locally advanced or metastatic urothelial carcinoma: results from the Real-world US PATRIOT-II Study. Clin Genitourin Cancer. 2024 Dec;22(6):102238. doi: 10.1016/j.clgc.2024.102238

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

Esterberg E, Iyer S, Nagar SP, Davis KL, Tannir NM. Real-world treatment patterns and clinical outcomes among patients with advanced renal cell carcinoma. Clin Genitourin Cancer. 2024 Apr;22(2):115-125.e. doi: 10.1016/j.clgc.2023.09.009

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

Bellmunt J, Chang J, Pavilack-Kirker M, Cappelleri JC, Costa N, Esterberg E, Kearney M, Hitchens A, Candrilli SD, Ajmera M. Evaluating real-world characteristics of patients with advanced urothelial carcinoma eligible for avelumab maintenance therapy: a multi-country retrospective medical chart review. Clin Genitourin Cancer. 2023 Aug;21(4):459-66. doi: 10.1016/j.clgc.2023.03.011

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

Stattin P, Westerberg M, Franck Lissbrant I, Hjalm-Eriksson M, Kjellman A, Ullen A, Vassilev Z, Sandstrom P, Weinrib R, Martinez D, Garcia de Albeniz Martinez X. Real world outcomes in patients with metastatic, castration-resistant prostate cancer treated with radium-223 in routine clinical practice in Sweden. Clin Genitourin Cancer. 2022 Sep 9;S1558-7673(22):00194-X. doi: 10.1016/j.clgc.2022.09.002

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

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

Hess G, Borker R, Fonseca E. Treatment patterns: targeted therapies indicated for first-line management of metastatic renal cell carcinoma in a real-world setting. Clin Genitourin Cancer. 2013 Jun;11(2):161-7. doi: 10.1016/j.clgc.2012.10.003

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.