Key patient-reported outcomes in children and adolescents with intoxication-type inborn errors of metabolism: an international Delphi-based consensus

Key patient-reported outcomes in children and adolescents with intoxication-type inborn errors of metabolism: an international Delphi-based consensus

Abstract Background Acute intoxication-type inborn errors of metabolism (IT-IEM) such as urea cycle disorders and non-acute IT-IEM such as phenylketonuria (PKU) and their treatment have a major impact on the life of affected children and families. Yet patients’ and parents’ perspectives on the burde...

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Bibliographic Details
Main Authors: Florin Bösch, Nina A. Zeltner, Matthias R. Baumgartner, Martina Huemer, Markus A. Landolt
Format: Article
Language:English
Published: BMC 2022-01-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Patient-reported outcomes
PROs
Patient-reported outcome measures
PROMs
Inborn errors of metabolism
Rare metabolic diseases
Online Access:https://doi.org/10.1186/s13023-022-02183-2

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