An autosomal-dominant childhood-onset disorder associated with pathogenic variants in VCP.
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Am J Hum Genet. 2023 Nov 2;110(11):1959-1975. doi: 10.1016/j.ajhg.2023.10.007. Epub 2023 Oct 25.
Am J Hum Genet. 2023.
PMID: 37883978
Free PMC article.