Congenital disorder of glycosylation – one size does not fit all: a parent’s perspective
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2022-08-22
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American English
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Sage
Abstract
This article is written by the parent of a child living with PMM2-congenital disorder of glycosylation (abbreviated to PMM2-CDG). It provides a parental perspective of the journey taken from diagnosis to present day and details the effect of off-label treatment with epalrestat.
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Cite As
Feinberg K. Congenital disorder of glycosylation - one size does not fit all: a parent's perspective. Ther Adv Rare Dis. 2022;3:26330040221118099. Published 2022 Aug 22. doi:10.1177/26330040221118099
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Therapeutic Advances in Rare Disease
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PMC
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Article
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Final published version