Estimating health state utilities for aromatic L-amino acid decarboxylase deficiency (AADCd) in the United States

Abstract Background AADCd is a rare neurometabolic disorder presenting in infancy. Children with AADCd have motor dysfunction and development delays that result in the need for lifelong care; quality of life is greatly impacted. Current characterizations of health-related quality of life and associa...

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Bibliografski detalji
Glavni autori: Berrin Monteleone, Katie Forster, Gin Nie Chua, Rongrong Zhang, Andrew Lloyd, Paul Castellano, Ioannis Tomazos
Format: Članak
Jezik:English
Izdano: BMC 2025-01-01
Serija:Health and Quality of Life Outcomes
Teme:
Online pristup:https://doi.org/10.1186/s12955-025-02335-5