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Glycogen Storage Disease Type Ia (2 companies)

Companies with an active program in Glycogen Storage Disease Type Ia.

Ticker Company Therapeutic areas Programs Lead stage Programs
BEAM Beam Therapeutics Inc. HematologyMetabolicRespiratory Rare 1 Phase 1 8
RARE Ultragenyx Pharmaceutical Inc. CardiovascularGI/HepatologyMetabolicNeurology Rare 1 Approved 12