Future Neurology

July 2008, Vol. 3, No. 4, Pages 375-384


Efficacy and safety of idebenone in the treatment of Friedreich ataxia: a review of early results and future prospects


Lisa S Friedman‌, Kimberly A Schadt‌, Robert B Wilson‌ & David R Lynch‌

University of Pennsylvania School of Medicine, Departments of Neurology & Pediatrics and, The Children’s Hospital of Philadelphia, PA 19104, USA. friedmanl@...

University of Pennsylvania School of Medicine, Departments of Neurology & Pediatrics and, The Children’s Hospital of Philadelphia, PA 19104, USA. schadt@...

University of Pennsylvania School of Medicine, Department of Pathology & Laboratory Medicine, PA, USA. wilsonr@...

Children´s Hospital of Philadelphia, Department of Neurology, Abramson Research Center, Room 502, 3615 Civic Center Blvd, PA 19104, USA. lynch@...

† Author for correspondence

Friedreich ataxia (FRDA) is an autosomal-recessive neurodegenerative disorder characterized by difficulty walking, dysarthria and absent reflexes. At present, no treatment or cure exists, but the pathophysiology of FRDA suggests that antioxidants and related compounds may be efficacious. Research in the field has focused on idebenone, a lipid-soluble synthetic short-chain analogue of the quinone coenzyme Q10. Idebenone protects mitochondrial membranes from oxidative damage in vitro and may facilitate electron transport within the mitochondrial electron transport chain. In FRDA, idebenone decreases cardiac hypertrophy, but the clinical significance of this finding remains unknown. Recent Phase II trials using higher doses of idebenone have suggested a dose-dependent improvement in neurologic symptoms. To confirm these results, a Phase III trial has begun in the USA with the goal of assessing the efficacy of idebenone for the treatment of neurologic dysfunction in FRDA.








The legacy of Marie Schlau: literature to help cure Friedreich's Ataxia

If you feel like reading an unputdownable novel while collaborating with a just and solidary cause, "The Legacy of Marie Schlau" is your book! 100% of all funds raised will be dedicated to medical research to find a cure for Friedreich's Ataxia, a neurodegenerative disease that affects mostly young people, shortening their life expectancy and confining them to a wheelchair.

The life of Marie Schlau, a German Jewish girl born in 1833 hides great unsolved mysteries: accidents, disappearances, enigmas, unknown diagnoses, disturbing murders, love, tenderness, greed, lies, death ... alternatively a different story unfolds every time and takes us closer to the present. Thus, there are two parallel stories unravelling, each in a different age and place, which surprisingly converge in a revelatory chapter.

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Research projects currently being financed by BabelFAmily

Currently, BabelFAmily is financing two promising research projects aimed at finding a cure for Friedreich's Ataxia. Whenever you make a donation to us or purchase a copy of "The legacy of Marie Schlau", this is where all funds raised will be devoted to:

1) Gene Therapy for Friedreich's Ataxia research project:

The project is the result of an initiative of Spanish people affected by this rare disease who are grouped in GENEFA in collaboration with the Spanish Federation of Ataxias and the BabelFAmily. The Friedreich’s Ataxia Research Alliance (FARA), one of the main patients’ associations in the United States now joins the endeavour.

2) Frataxin delivery research project:
The associations of patients and families Babel Family and the Asociación Granadina de la Ataxia de Friedreich (ASOGAF) channel 80,000 euros of their donations (50% from each organisation) into a new 18-month project at the Institute for Research in Biomedicine (IRB Barcelona). The project specifically aims to complete a step necessary in order to move towards a future frataxin replacement therapy for the brain, where the reduction of this protein causes the most damage in patients with Friedreich’s Ataxia.

The study is headed by Ernest Giralt, head of the Peptides and Proteins Lab, who has many years of experience and is a recognised expert in peptide chemistry and new systems of through which to delivery drugs to the brain, such as peptide shuttles—molecules that have the capacity to carry the drug across the barrier that surrounds and protects the brain. Since the lab started its relation with these patients’ associations in 2013*, it has been developing another two projects into Friedrich’s Ataxia.



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