Primary coenzyme Q10 deficiency
First line
High-dose CoQ10 (10–30 mg/kg/day, in adults up to 1,200–2,400 mg daily) is the only disease-modifying approach to the rare inherited primary CoQ10 deficiency. Early therapy in children prevents progression of encephalopathy and nephrotic syndrome; in adults it stabilises cerebellar ataxia. GeneReviews and international mitochondrial disease guidelines support this use.
Used in specialised centres with confirmed genetic diagnosis; not for self-prescription.