Development of the Dutch translational knowledge agenda for inherited metabolic diseases

Development of the Dutch translational knowledge agenda for inherited metabolic diseases

Abstract Background Inherited metabolic diseases (IMDs) may have considerable implications for patients and their families. Despite their individual rarity, covering a spectrum of over 1800 distinct diseases, the diseases collectively exert a significant impact, with often lifelong disabilities. The...

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Main Authors: I. J. Hieltjes, J. H. van derLee, M. C. Groenendijk, G. vanHaaften, P. M. vanHasselt, R. J. Lunsing, G. J. J. vanProoijen, E. M. deRuiter, F. J. vanSpronsen, N. M. Verhoeven‐Duif, A. deVreugd, M. Wagenmakers, H. Zweers, H. Dekker, H. R. Waterham, C. D. vanKarnebeek, R. J. A. Wanders, R. A. Wevers
Format: Article
Language:English
Published: Wiley 2025-01-01
Series:JIMD Reports
Subjects:
gene therapy
knowledge agenda
metabolomics
natural course
pathophysiology
patient and public involvement
Online Access:https://doi.org/10.1002/jmd2.12455
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https://doi.org/10.1002/jmd2.12455

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