Improvements with burosumab treatment in an early access programme for adults with X-linked hypophosphataemia: A case series of three patients

Improvements with burosumab treatment in an early access programme for adults with X-linked hypophosphataemia: A case series of three patients

X-linked hypophosphataemia (XLH) is a life-long phosphate-wasting disorder that causes skeletal deformities, pain, stiffness, and fatigue and impairs quality of life. Burosumab was approved for use in adults in 2020. We describe three adults with persistent XLH symptoms who received burosumab treatm...

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Bibliographic Details
Main Authors: Julia Day, Chandrin Jayatilleke, Matthew Roy
Format: Article
Language:English
Published: Elsevier 2024-12-01
Series:Bone Reports
Subjects:
Burosumab
Patient-reported outcome
Case series
Early access
X-linked hypophosphatemia
Online Access:http://www.sciencedirect.com/science/article/pii/S2352187224000810
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