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NGLY1 deficiency: Novel patient, review of the literature and diagnostic algorithm

OBJECTIVES: Together with the lysosomal storage diseases, NGLY1 deficiency is a congenital disorder of deglycosylation (NGLY1‐CDDG). Since the first report in 2012, 26 patients have been described. All but one were diagnosed by exome or genome sequencing; the remaining one was identified by finding...

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Bibliografiske detaljer
Udgivet i:JIMD Rep
Main Authors: Lipiński, Patryk, Bogdańska, Anna, Różdżyńska‐Świątkowska, Agnieszka, Wierzbicka‐Rucińska, Aldona, Tylki‐Szymańska, Anna
Format: Artigo
Sprog:Inglês
Udgivet: John Wiley & Sons, Inc. 2020
Fag:
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC7012742/
https://ncbi.nlm.nih.gov/pubmed/32071843
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/jmd2.12086
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