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

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 burdens of IT...

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Detalles Bibliográficos
Autores principales: Bösch, Florin, Zeltner, Nina A., Baumgartner, Matthias R., Huemer, Martina, Landolt, Markus A.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2022
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8800290/
https://www.ncbi.nlm.nih.gov/pubmed/35093149
http://dx.doi.org/10.1186/s13023-022-02183-2

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