IGF2-tagging of GAA promotes full correction of murine Pompe disease at a clinically relevant dosage of lentiviral gene therapy - ScienceDirect
Drug induced exocytosis of glycogen in Pompe disease - ScienceDirect
Immunomodulatory, liver depot gene therapy for Pompe disease - ScienceDirect
Diagnosis and treatment of late-onset Pompe disease in the Middle East and North Africa region: consensus recommendations from an expert group | BMC Neurology | Full Text
GK for UPSC & TSPSC on X: "Pompe Disease Source: Indian Express https://t.co/dEc9GicYf1" / X
Newborn Screening - Pompe Disease - EveryLife Foundation for Rare Diseases