Treatment preferences among individuals with primary hyperoxaluria type 1 (PH1): a real-world study

Treatment preferences among individuals with primary hyperoxaluria type 1 (PH1): a real-world study

Abstract Background Primary hyperoxaluria type 1 (PH1) is a rare genetic disorder causing excessive oxalate production, damaging kidneys and other organs. Nedosiran, launched in the U.S. for individuals with PH1 (≥ 9 years of age; estimated glomerular filtration rate [eGFR] ≥ 30 mL/min/1.73 m2), can...

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Main Authors: David S. Goldfarb, Jing Voon Chen, Rebekah Zincavage, Brad Padilla, Matthew Sussman, Sandra Salem, Frank Modersitzki
Format: Article
Language:English
Published: BMC 2025-05-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Primary hyperoxaluria
Real-world study
Patient survey
Treatment preferences
Online Access:https://doi.org/10.1186/s13023-025-03738-9
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