Abstract
The authors carried out a 1-year, randomized, placebo-controlled trial of idebenone in 29 patients with Friedreich ataxia. They found significant reductions of interventricular septal thickness and left ventricular mass in the idebenone group vs the placebo group, with no improvement in other heart ultrasound measures or neurologic condition. The absolute cardiac changes were modest, but the findings suggest that larger trials should assess whether idebenone reduces ventricular hypertrophy in patients with Friedreich ataxia.
Publication types
-
Clinical Trial
-
Randomized Controlled Trial
-
Research Support, Non-U.S. Gov't
MeSH terms
-
Adolescent
-
Adult
-
Benzoquinones / pharmacology
-
Benzoquinones / therapeutic use*
-
Cardiomyopathy, Hypertrophic / drug therapy
-
Cardiomyopathy, Hypertrophic / etiology
-
Child
-
Erythrocytes / chemistry
-
Female
-
Frataxin
-
Free Radical Scavengers / pharmacology
-
Free Radical Scavengers / therapeutic use*
-
Free Radicals
-
Friedreich Ataxia / complications*
-
Friedreich Ataxia / genetics
-
Humans
-
Hypertrophy, Left Ventricular / drug therapy*
-
Hypertrophy, Left Ventricular / etiology
-
Iron-Binding Proteins / genetics
-
Iron-Binding Proteins / physiology
-
Male
-
Mitochondria / metabolism
-
Oxidative Stress
-
Prospective Studies
-
Protoporphyrins / blood
-
Ubiquinone / analogs & derivatives
Substances
-
Benzoquinones
-
Free Radical Scavengers
-
Free Radicals
-
Iron-Binding Proteins
-
Protoporphyrins
-
Ubiquinone
-
protoporphyrin IX
-
idebenone