Caregiver burden, and parents' perception of disease severity determine health-related quality of life in paediatric patients with intoxication-type inborn errors of metabolism.

"Declaration of Competing Interest The authors of this manuscript declare no competing interests but disclose the following: F. Bösch, M.A. Landolt, S. Fernandez, P. Forny, M. Gautschi, S.C. Grünert, C. Horvath, D. Karall, D. Lampis, M. Rohrbach, and Gabor Szinnai report that they have nothing to disclose. M. R. Baumgartner received a research fund from Nutricia Metabolics and is a member of the clinical advisory boards of Hemoshear and Moderna. J. Häberle has received a research grant from Nutricia Metabolics and is collaborating in research with Recordati Rare Diseases. S. Scholl-Bürgi has received travel support from Nutricia Metabolics. M. Huemer has received research grants from Nutricia Metabolics and consultancy honoraria from Aeglea, Nutricia Metabolics, Sanofi, Shire, SOBI, and Orphan Europe."

"This publication was enabled by research grants from the Vontobel Foundation and Nutricia Metabolics. M.A.L., M.R.B. and J.H. are supported by the University Research Priority Program of the 10.13039/501100006447University of Zurich (URPP) ITINERARE—Innovative Therapies in Rare Diseases. M.R.B. is supported by the 10.13039/501100001711Swiss National Science Foundation [31003A_175779]. We thank all participating parents and patients for their contribution."