Glycogen storage disease due to glucose-6-phosphatase deficiency Advocacy Hub | UniteRare

At a Glance

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Regulatory Watchboard

2 events

Dec 2024A Phase 1/2, Dose-Exploration Study to Evaluate the Safety and Efficacy of BEAM-301 in Patients With Glycogen Storage Disease Type Ia (GSDIa)

Beam Therapeutics Inc. — PHASE1, PHASE2

TrialRECRUITING

Nov 2024Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program

Ultragenyx Pharmaceutical Inc

TrialRECRUITING

Data from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

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FINANCIAL LANDSCAPE SUMMARY

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Clinical Trial Landscape

2 active trials

1Unknown

1PHASE1, PHASE2

2Total recruiting

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