In vitro and in vivo studies indicate that VA activates endogenous CoQ synthesis and rescues the phenotype in COQ6 deficiency, while β-RA reduces the DMQ/COQ ratio, and thus might be a suitable approach in patients with defects of CoQ biosynthesis, which cause accumulation of DMQ, as mutations in COQ9, COQ7, or COQ4. The gene discussed is COQ7; the disease is hyperinsulinemic hypoglycemia, familial, 4.