Last update 04 Apr 2026

Glygogen Storage Disease Type 1a (Genethon)

Overview

Basic Info

Drug Type
Gene therapy
Synonyms-
Target
G6PC1
Action
stimulants
Mechanism
G6PC1 stimulants(Glucose-6-phosphatase stimulants)
Therapeutic Areas
Congenital DisordersEndocrinology and Metabolic DiseaseOther Diseases
Active Indication
Hepatorenal Form of Glycogen Storage Disease
Inactive Indication-
Originator Organization
GenethonGenethon
Active Organization
GenethonGenethon
Inactive Organization-
License Organization-
Drug Highest PhasePreclinical
First Approval Date-
Regulation-

Related

100 Clinical Results associated with Glygogen Storage Disease Type 1a (Genethon)
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100 Translational Medicine associated with Glygogen Storage Disease Type 1a (Genethon)
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100 Patents (Medical) associated with Glygogen Storage Disease Type 1a (Genethon)
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100 Deals associated with Glygogen Storage Disease Type 1a (Genethon)
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R&D Status

10 top R&D records.
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IndicationHighest PhaseCountry/LocationOrganizationDate
Hepatorenal Form of Glycogen Storage DiseasePreclinical
France
GenethonGenethon
01 May 2025
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Clinical Result

Indication
Phase
Evaluation
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Approval

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Regulation

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