Idebenone treatment in Friedreich patients: one-year-long randomized placebo-controlled trial.
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.
Mariotti
C
C
Division of Biochemistry and Genetics, Carlo Besta National Neurological Institute-IRCCS, Milan, Italy.
Solari
A
A
Torta
D
D
Marano
L
L
Fiorentini
C
C
Di Donato
S
S
D000293:Adolescent; D000328:Adult; D016227:Benzoquinones / Q000494:pharmacology / Q000627:therapeutic use*; D002312:Cardiomyopathy, Hypertrophic / Q000188:drug therapy / Q000209:etiology; D002648:Child; D004912:Erythrocytes / Q000737:chemistry; D005260:Female; D016166:Free Radical Scavengers / Q000494:pharmacology / Q000627:therapeutic use*; D005609:Free Radicals; D005621:Friedreich Ataxia / Q000150:complications* / Q000235:genetics; D006801:Humans; D017379:Hypertrophy, Left Ventricular / Q000188:drug therapy* / Q000209:etiology; D033862:Iron-Binding Proteins / Q000235:genetics / Q000502:physiology; D008297:Male; D008928:Mitochondria / Q000378:metabolism; D018384:Oxidative Stress; D011446:Prospective Studies; D011524:Protoporphyrins / Q000097:blood; D014451:Ubiquinone / Q000031:analogs & derivatives; D000096923:Frataxin
D016227:Benzoquinones; D016166:Free Radical Scavengers; D005609:Free Radicals; D033862:Iron-Binding Proteins; D011524:Protoporphyrins; D014451:Ubiquinone; D000096923:Frataxin; C028025:protoporphyrin IX; C036619:idebenone