Newborn screening of primary carnitine deficiency: clinical and molecular genetic characteristics

Abstract Background Primary carnitine deficiency (PCD) is a rare autosomal recessive fatty acid oxidation disorder caused by variants in the SLC22A5 gene, with its prevalence and the spectrum of mutations in SLC22A5 varying across races and regions. This study aimed to analyze the clinical and genet...

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Bibliographic Details
Main Authors: Haili Hu, Qingqing Ma, Yan Wang, Wangsheng Song, Hongyu Xu
Format: Article
Language:English
Published: BMC 2025-03-01
Series:Italian Journal of Pediatrics
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Online Access:https://doi.org/10.1186/s13052-025-01911-1
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