Dramatic improvement in mitochondrial cardiomyopathy following treatment with idebenone.

Mitochondrial Disease Clinic,

J Inherit Metab Dis 2001 Feb; 24(1): 28-34


Idebenone has been shown to improve cardiac function in patients with Friedreich ataxia and a deficiency of respiratory chain complexes I-III. We describe a woman with severe combined right and left heart failure due to a mitochondrial cardiomyopathy.  The patient underwent an endomyocardial biopsy as part of an evaluation for cardiac transplantation. It showed severely decreased respiratory complex activities dependent on CoQ, pointing to CoQ depletion. Following 
treatment there was a dramatic improvement in her clinical status with resolution of the heart failure.

Successful idebenone therapy of hypertrophic cardiomyopathy in Friedreich’s ataxia

Gottsegen Gyorgy Orszagos Kardiologiai Intezet,
Orv Hetil. 2003 Jun 22; 144(25):1241-4


Severe hypertrophic cardiomyopathy in a 15 years old child with Friedreich ataxia was treated with idebenone on the basis of a preliminary study reported in the literature. After 3 months of reatment the muscle thickness and mass and idices of diastolic function on echocardiogram and ischaemic signs on ECG changed significantly.

CONCLUSION: Our data proves the preliminary results, so idebenone treatment is very effective in the disease, where a deficiency of frataxin is involved in the regulation of mitochondral iron content which is responsible for myocardial injury. We suggest the widespread use of idebenone to treat patients with Friedreich ataxia and hypertrophic cardiomyopathy to improve the fatal prognosis of this type of cardiomyopathy.