Rare Diseases

Joly F, DasMahapatra P, Su J, DiBenedetti D, Kosa K, Hill Q. Development of a cold Agglutinin disease-specific patient-reported outcome symptom measure. Poster presented at the 2021 63rd American Society of Hematology (ASH) Annual Meeting & Exposition; December 13, 2021. [abstract] Blood. 2021 Nov 23; 138(supplement 1):4057. doi: 10.1182/blood-2021-147199


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


Bektas M, Copley-Merriman C, Khan S, Sarda SP, Shammo JM. Paroxysmal nocturnal hemoglobinuria: current treatments and unmet needs. J Manag Care Spec Pharm. 2020 Dec;26(12-b Suppl):S14-20. doi: 10.18553/jmcp.2020.26.12-b.s14


Bektas M, Copley-Merriman C, Khan S, Sarda SP, Shammo JM. Paroxysmal nocturnal hemoglobinuria: role of the complement system, pathogenesis, and pathophysiology. J Manag Care Spec Pharm. 2020 Dec;26(12-b Suppl):S3-8. doi: 10.18553/jmcp.2020.26.12-b.s3


Bektas M, Copley-Merriman C, Khan S, Sarda SP, Shammo JM. Paroxysmal nocturnal hemoglobinuria: patient journey and burden of disease. J Manag Care Spec Pharm. 2020 Dec;26(12-b Suppl):S8-S14. doi: 10.18553/jmcp.2020.26.12-b.s8


Goyal RK, Jain P, Nagar SP, Le H, Kabadi SM, Davis K, Kaye JA, Du XL, Wang M. Real-world evidence on survival, adverse events, and health care burden in Medicare patients with mantle cell lymphoma. Leuk Lymphoma. 2021 Jun;62(6):1325-34. doi: 10.1080/10428194.2021.1919662


Wu B, Morland K, Classi P, Sketch MR, Nelson A, Goyal RK, Davis KL, Oudiz R. Economic burden of patients with pulmonary arterial hypertension prior to initiation of treprostinil: a retrospective analysis of US claims. Poster presented at the ATS International 2021 Conference; May 14, 2021.


Davis A, Jiang J, Talbird S, Turpin RS, Mellott CE, Boules M, Wojtowicz AM, Fan T. Pharmacological costs for the treatment of eosinophilic esophagitis in the USA. Poster presented at the 2021 Virtual Digestive Disease Week (DDW) Conference; May 21, 2021. Previously presented at the 2021 AMCP Conference.


Davis AE, Jiang J, Talbird SE, Turpin R, Mellott CE, Boules M, Wojtowicz AM, Fan T. Pharmacological costs for the treatment of eosinophilic esophagitis in the USA. Poster presented at the 2021 Virtual AMCP; April 2021.


Mader G, Mladsi DM, Zhou X, Sanon M, Wang J, Willey CJ, Seliger SL. Mortality risk in elderly ESRD patients with Autosomal Dominant Polycystic Kidney Disease (ADPKD): study findings using data from the United States Renal Data System. Poster presented at the 2020 Virtual American Society of Nephrology Kidney Week Conference; October 22, 2020. [abstract] J Am Soc Nephrol. 2020 Oct; 31:503-4.


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