Rare Diseases

Ciepielewska M, Hagan M, Houghton K, Hitchens A, Ansquer V, Silver SM. Real-world treatment patterns and healthcare resource utilization among patients diagnosed with erythropoietic protoporphyria and x-linked protoporphyria: results from a US electronic medical records study. Poster presented at the ISPOR 2022 Conference; May 17, 2022. Washington, DC. [abstract] Value Health. 2022 Jun; 25(6 S1).


Whalley D, Belongie K, Frangiosa T, Krasa H, Mladsi DM, Twiss J, Wolowacz S. Understanding the patient experience of erythropoietic protoporphyria and x-linked protoporphyria: a qualitative study. Poster presented at the ISPOR 2022 Conference; May 15, 2022. Washington, DC. [abstract] Value Health. 2022 Jun; 25(6 S1).


Graham CN, Watson C, Barlev A, Stevenson M, Dharnidharka VT. Mean lifetime survival estimates following solid organ transplantation in the US and UK. J Med Econ. 2022 Jan;25(1):230-7. doi: 10.1080/13696998.2022.2033050


Anderson-Smits C, Mitchell S, Hawe E, Hartley L. Use of intravenous immunoglobulin in patients with chronic inflammatory demyelinating polyneuropathy: a systematic literature review. Poster presented at the 2022 Peripheral Nerve Society (PNS) Annual Meeting; May 14, 2022. Miami, FL.


Abstract not available at this time.

Anderson-Smits C, Park M, Bell J, Mitchell S, Hartley L, Hawe E. Subcutaneous immunoglobulin use in immunoglobulin-naïve patients with primary immunodeficiency: a systematic review. Immunotherapy. 2022 Apr;14(5):373-87. doi: 10.2217/imt-2021-0265


AIMS: Identify and describe published literature on the use of subcutaneous immunoglobulin (SCIG) as initial immunoglobulin (IG)-replacement therapy for patients with primary immunodeficiency diseases (PID).

Yee KS, Alexanderian D, Hamm L, Martin S, Olayinka-Amao B, Whiteman DAH. Clinical investigator perspectives on the effects of intrathecal idursulfase-IT treatment in children with neuronopathic mucopolysaccharidosis type II. Poster presented at the 2022 18th Annual World Symposium; February 7, 2022. San Diego, CA.


Anderson S, Talbird SE, Fishman J, Mody-Patel N, Sarda SP. Budget impact of pegcetacoplan, a complement c3 inhibitor, for the treatment of paroxysmal nocturnal hemoglobinuria in US adults. Poster presented at the Virtual ISPOR Europe 2021; November 30, 2021. [abstract] Value Health. 2021 Dec; 24(12):S2.


Lunan M, Hartley L, Pearson I, Doyle S. Targeted literature review of the burden of illness in late-stage Duchenne Muscular Dystrophy (DMD). Poster presented at the Virtual ISPOR Europe 2021; November 3, 2021. [abstract] Value Health. 2021 Dec; 24(12):S2.


Anderson S, Talbird S, Fishman J. Cost per responder analysis for pegcetacoplan and eculizumab in the treatment of adults with paroxysmal nocturnal hemoglobinuria. Blood. 2021;138(Suppl 1):4956. doi: 10.1182/blood-2021-153190


Gold BD, Goodwin B, Davis K, Sweeney C, Ziemiecki R, Jiang J, Fan T, Boules M, Desai NK, Katzka DA. Patient satisfaction and adherence to corticosteroids in adolescents and adults with eosinophilic esophagitis in a real-world setting in the US. Poster presented at the 2021 Annual Scientific Meeting & Postgraduate Course (ACG); October 24, 2021. Las Vegas, NV. [abstract] Am J Gastroenterol. 2021 Oct; 116:S177.


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