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Clinicians urge adaptive emergency plans for glycogen storage disease; multiple gene- and RNA-therapy trials under way
Summary
Pediatric endocrinologist Rebecca Wolman briefed the RDAC on emergency management for glycogen storage diseases and summarized early-stage gene, RNA and gene-editing trials underway at Connecticut sites.
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…
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