Clinical, biochemical, and genetic analysis of a Chinese Han pedigree with holocarboxylase synthetase deficiency: a case report

中国汉族全羧化酶合成酶缺乏症家系的临床、生化和遗传分析:一例病例报告

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Background

Holocarboxylase synthetase (HLCS) deficiency is a rare inborn disorder of biotin metabolism, which

Conclusions

The Chinese proband carries the reported Arg508Trp variant, the novel 2-bp frameshift mutation c.1006_1007delGA (p.Glu336Thrfs*15), which expands the mutational spectrum of the HLCS gene, and the novel heterozygous mutation c.638_642delAACAC (p.His213Profs*4), which expands the mutational spectrum of the BTD gene. Furthermore, reversible hearing damage is rarely reported in patients with HLCS deficiency, which deserves further

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