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… 

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References

SHOWING 1-10 OF 11 REFERENCES

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.

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 offerrochelatase leading to a state of ineffective, persistent erythropoiesis.

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.

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.

The Metabolic and Molecular Bases of Inherited Disease (Scriver, C. R., Beaudet, A. L., Sly, W. S., Valle, D., Childs, B., Kinzler, K. W., and Vogelstein, B., eds., 8th ed., McGraw-Hill, New-York, 2001, 7012 p., $550.00)

The current eighth edition of the “Nauka/Interperiodica” is significant ly different from its predecessors, with a large international group of authors, with the total number of over 500 contributors.

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.

A micromethod for free erythrocyte porphyrins: the FEP test.

  • S. Piomelli
  • Medicine
    The Journal of laboratory and clinical medicine
  • 1973
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.

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.