The patient experience of CHAPLE disease: results from interviews conducted as part of a clinical trial for an ultra-rare condition

The patient experience of CHAPLE disease: results from interviews conducted as part of a clinical trial for an ultra-rare condition

Abstract Background CD55 deficiency with hyper-activation of complement, angiopathic thrombosis, and protein-losing enteropathy (CHAPLE) disease is a newly identified condition with an estimated worldwide prevalence of < 100 patients. Patient interviews can ensure that what is important to patien...

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Bibliographic Details
Main Authors: Leighann Litcher-Kelly, Ahmet Ozen, Sarah Ollis, Hagit Baris Feldman, Andrew Yaworsky, Paolo Medrano, Voranush Chongsrisawat, Lorah Perlee, Marisa Walker, Sharanya Pradeep, Diane M. Turner-Bowker, Alina Kurolap, Orly Eshach Adiv, Michael J. Lenardo, Olivier A. Harari, Jessica J. Jalbert
Format: Article
Language:English
Published: BMC 2025-02-01
Series:Orphanet Journal of Rare Diseases
Subjects:
CHAPLE
Within-trial interviews
Pozelimab
HRQoL
Online Access:https://doi.org/10.1186/s13023-024-03436-y
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