Rebecca "Reba" Wolman, a pediatric endocrinologist, told the council that individuals with hepatic glycogen storage diseases (GSDs) face acute emergency risks that differ from common hypoglycemia protocols and described ongoing investigational therapies being studied at Connecticut clinical sites.

The nut graf: Wolman said standard hypoglycemia responses (glucagon, oral sugar, single bolus dextrose) may be harmful in some GSD patients and stressed the need for hospital emergency plans and EMR advisories; she also summarized three classes of investigational products—AAV gene therapy, lipid-nanoparticle RNA therapy and nucleotide-editing approaches—being studied in early-stage trials.

Wolman described clinical differences relevant to ER care: "Glucagon can be given intranasally, IV...that is the correct first step for people with diabetes. On the other side of the screen, actually giving that to somebody with GSD 1a causes very severe lactic acidosis," she said, and added that continuous dextrose infusion is often the appropriate therapy for these patients. She noted that existing institutional work-arounds include Epic best-practice advisories and standardized emergency letters entered as notes so they are visible across institutions.

Wolman summarized investigational treatments in GSD 1a: an AAV-based nonintegrating in vivo gene therapy (Ultragenyx), an RNA lipid-nanoparticle therapy (Moderna) and an adenine base-editor gene-editing approach (Beam Therapeutics). She said Connecticut sites participated in early infusions for these multinational studies and presented early-phase signals suggesting reduced cornstarch dependence and improvements in fasting tolerance in small cohorts; she cautioned that these are investigational and early results.

Ending: Wolman said emergency-protocol alignment across hospitals remains a priority and that clinical-trial activity in Connecticut offers potential pathways toward disease-modifying therapies, while stressing that these products remain experimental and require long-term follow-up.