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Clinical outcomes of two patients with a novel pathogenic variant in ASNS: response to asparagine supplementation and review of the literature

Asparagine synthetase deficiency (ASNSD, OMIM #615574) is a rare autosomal recessive neurometabolic inborn error that leads to severe cognitive impairment. It manifests with microcephaly, intractable seizures, and progressive cerebral atrophy. Currently, there is no established treatment for this co...

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Bibliografiske detaljer
Udgivet i:Hum Genome Var
Main Authors: Sprute, Rosanne, Ardicli, Didem, Oguz, Kader Karli, Malenica-Mandel, Anna, Daimagüler, Hülya-Sevcan, Koy, Anne, Coskun, Turgay, Wang, Haicui, Topcu, Meral, Cirak, Sebahattin
Format: Artigo
Sprog:Inglês
Udgivet: Nature Publishing Group UK 2019
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Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC6531480/
https://ncbi.nlm.nih.gov/pubmed/31123592
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41439-019-0055-9
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