glycogen storage disease

(redirected from glycogen storage disease type Ia)
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glycogen storage disease

[′glī·kə·jən ′stȯr·ij di‚zēz]
References in periodicals archive ?
Glycogen storage disease type Ia: frequency and clinical course in Turkish children.
(NASDAQ: RARE) fell 10.9% to $45.69 after the company reported data from Phase 1/2 study of DTX401 gene therapy in glycogen storage disease type Ia.
Food and Drug Administration has cleared the Investigational New Drug application for DTX401, an adeno-associated virus vector based gene therapy for the treatment of glycogen storage disease type Ia. Enrollment in the Phase 1/2 study is expected to begin in the first half of 2018, with data from the first cohort expected in the second half of 2018.

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