Glycogen Storage Disease Type Iv
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CLINICAL_TRIALS research · 2025-05-28Clinical Trial Update: GBE Deficiency (GSD IV and APBD) Natural History Study
Observational natural history study of GBE deficiency including Glycogen Storage Disease Type IV and Adult Polyglucosan Body Disease, curren…
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CLINICAL_TRIALS trial_result · 2018-03-05Clinical Trial Update: Triheptanoin Treatment Trial for Patients With Adult Polyglucosan Body Disease [Phase 2]
Phase 2 trial of triheptanoin for Adult Polyglucosan Body Disease has completed.