Idebenone treatment in Friedreich’s ataxia

  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},
  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
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 damageExpand
Monitoring cardiac function during idebenone therapy in Friedreich's ataxia.
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
Low-dose idebenone treatment in Friedreich’s ataxia with and without cardiac hypertrophy
A retrospective analysis of a cohort of 35 patients with confirmed molecular diagnosis of Friedreich’s ataxia, treated with idebenone 5 mg/kg/day for up to five years finds an increase of interventricular septum and posterior wall thickness in the group without LVH before treatment and no change before treatment. Expand
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 andExpand
Patient‐reported outcomes in Friedreich’s ataxia after withdrawal from idebenone
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
Progress in the treatment of Friedreich ataxia.
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
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
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 proteinExpand
Co‐enzyme Q10 and idebenone use in Friedreich's ataxia
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


Idebenone in patients with Friedreich ataxia
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
Idebenone and reduced cardiac hypertrophy in Friedreich's ataxia
There is a good case for giving idebenone continuously in a dose of 5–10 mg/kg/day in patients with Friedreich's ataxia at the onset of hypertrophic cardiomyopathy, as the drug has no serious side effects. Expand
Erythrocyte protoporphyrin levels in patients with Friedreich's and other ataxias.
The finding of elevated FEP may indicate a relative heme deficiency in ataxia due to inhibition of ferrochelatase leading to a state of ineffective, persistent erythropoiesis. Expand
Effect of idebenone on cardiomyopathy in Friedreich's ataxia: a preliminary study
The in-vitro data suggest that both iron chelators and antioxidant drugs that may reduce iron are potentially harmful in patients with Friedreich's ataxia and that idebenone protects heart muscle from iron-induced injury. Expand
Idebenone. A review of its pharmacodynamic and pharmacokinetic properties, and therapeutic use in age-related cognitive disorders.
In view of the lack of a proven agent to limit or halt the progression of dementia in the elderly, idebenone may warrant consideration in patients with mild cognitive dysfunction on the basis of preliminary evidence of predominantly mild improvement of functional status in some patients and good tolerability. Expand
Disposition of idebenone (CV-2619), a new cerebral metabolism improving agent, in rats and dogs.
In rats given 14C-CV-2619 orally or intravenously, 14C was distributed widely in tissues, with relatively high concns. Expand
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0006 2979/02/6705 0611$27.00 ©2002 MAIK “Nauka/Interperiodica” The previous seven editions of this book are well known to the large audience of specialists all over the world. Published in 1960, theExpand
New Ultrasound Methods to Quantify Regional Myocardial Function in Children with Heart Disease
Preliminary data is presented on the potential clinical value of ultrasonic regional SR and e imaging in children and the close interrelation of these new regional function parameters with an alternative approach to quantification: the measurement of local cyclical changes in integrated backscatter levels. Expand
A micromethod for free erythrocyte porphyrins: the FEP test.
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The micromethod (FEP test) appears to be a promising tool to screen children for lead poisoning and compare favorably with those of a classical technique. Expand
Echocardiographic Determination of Left Ventricular Mass in Man: Anatomic Validation of the Method
The best method for LVM-E identified combined cube function geometry with a modified convention for determination of left ventricular internal dimension (LVID), posterior wall thickness (PWT), and interventricular septal thickness (IVST), which excluded the thickness of endocardial echo lines from wall thicknesses and included the thickness in LVID. Expand