Idebenone treatment in Friedreich’s ataxia

@article{Buyse2003IdebenoneTI,
  title={Idebenone treatment in Friedreich’s ataxia},
  author={Gunnar M. Buyse and Luc Mertens and Giovanni Di Salvo and I Matthijs and Frank Weidemann and B{\'e}n{\'e}dicte Eyskens and Willy Goossens and Nathalie Goemans and George R. Sutherland and Johan L. K. Van Hove},
  journal={Neurology},
  year={2003},
  volume={60},
  pages={1679 - 1681}
}
The authors report 1-year prospective data on eight patients with Friedreich ataxia. Idebenone did not halt the progression of ataxia. At the end of therapy, cardiac ultrasound demonstrated significant reduction of cardiac hypertrophy in six of eight patients. Cardiac strain and strain rate imaging showed that the reduction of hypertrophy is preceded by an early and linear improvement in cardiac function. Idebenone reduced erythrocyte protoporphyrin IX levels in five of six patients with… Expand
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Neurological eff ects of high-dose idebenone in patients with Friedreich ’ s ataxia : a randomised , placebo-controlled trial
Background Friedreich’s ataxia (FA) is a progressive, multisystem, degenerative disorder caused by a reduction in frataxin. Loss of frataxin results in mitochondrial dysfunction and oxidative damage… Expand
Monitoring cardiac function during idebenone therapy in Friedreich's ataxia.
TLDR
The present article reviews the clinical features of Friedreich's ataxia, imaging techniques used to diagnose, follow and monitor therapy which aimed to revert FA cardiomyopathy, and studies on Idebenone treatment which showed rather conflicting results. Expand
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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… Expand
Patient‐reported outcomes in Friedreich’s ataxia after withdrawal from idebenone
TLDR
The aim was to capture subjective experiences of symptoms such as fatigue, which can be difficult to measure with questionnaires or semi‐quantitative scales, particularly in chronic, slowly progressive conditions. Expand
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TLDR
Research into treatment of FRDA has advanced considerably over the last two decades since the genetic cause was identified, and current proposed treatment strategies include decreasing oxidative stress, increasing cellular frataxin, improving mitochondrial function as well as modulating fratAXin controlled metabolic pathways. Expand
Cardiomiopatía hipertrófica en ataxia de Friedreich. Presentación de dos casos
TLDR
The cases here described highlight the importance of early screening and identification of systemic complications, specifically cardiac disease, in patients with Friedreich’s ataxia. Expand
Management and therapy for cardiomyopathy in Friedreich’s ataxia
TLDR
The present artcle reviews the molecular basis of the disease, the clinical features of cardiomyopathy in Friedreich’s ataxia and the upcoming therapies. Expand
Co-enzyme Q 10 and idebenone use in Friedreich ’ s ataxia
Friedreich’s ataxia is a debilitating progressive neurodegenerative disease associated with cardiomyopathy and other features. The underlying cause is a deficiency of the mitochondrial protein… Expand
Co‐enzyme Q10 and idebenone use in Friedreich's ataxia
TLDR
This article reviews trials of the two most important agents, namely co‐enzyme Q10 and idebenone in Friedreich's ataxia, and assesses their efficacy over the last 15 years. Expand
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P magnetic resonance spectroscopy demonstrated mitochondrial impairment in vivo in skeletal muscle of all FA patients, but no recovery with idebenone, and Echocardiography did not confirm a preliminary study reporting improvement of FA-associated cardiomyopathy with Idebenone. Expand
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