Drug Repurposing

A Cholesterol Drug Reopens a Door for a Rare Glycosylation Disorder
September 4, 2026

A Cholesterol Drug Reopens a Door for a Rare Glycosylation Disorder

Congenital disorders of glycosylation are a family of more than two hundred inherited diseases in which protein glycosylation is disrupted, leading to diverse multisystem symptoms and, too often, no approved therapies. SRD5A3-CDG is among the rarest subtypes, with approximately 60 cases reported to date, and causes ataxia, developmental delay, and visual impairment through a defect […]

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