Two recent case reports show that coenzyme Q10 (ubiquinone) therapy can be an effective treatment for rare, previously untreatable respiratory chain dysfunction.
French researchers reported on two siblings, a brother and sister, who were both affected by multiple respiratory enzyme deficiency. Both children were born apparently normal, but their motor abilities, vision, hearing and, eventually, kidney function became progressively worse over time.
Blood tests revealed widespread coenzyme Q10 deficiency and both children were put on a 5 mg/kg daily dose of oral ubidecarenone. After three years of therapy, both children showed substantial improvement in their conditions. After just two months of therapy, the boy, who was wheelchair bound, could stand and walk unaided. His motor skills also improved and a cataract in his right eye disappeared. His sister was able to speak more clearly, write, catch a ball and interact with her environment more effectively (Lancet, 2000; 356: 391-5).