Are cardiac problems in FA somewhat under-recognized?
Almost all the publications on this disease are on the neurologic impact, but heart disease is what kills people. Unfortunately,
Voices of Friedreich's Ataxia (FA)
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Almost all the publications on this disease are on the neurologic impact, but heart disease is what kills people. Unfortunately,
Treatments for Friedreich’s ataxia (F A) generally target specific symptoms rather than the disease itself. Fortunately, F A’s most life-threatening
One approach to treating F A would be to supply functional frataxin genes or protein molecules to the person with
HDAC inhibition The biotechnology company Repligen has received MDA support for the development of an experimental treatment for FA known
Another approach to treating FA involves inserting new frataxin protein molecules. Recent research has shown that the frataxin protein can
Iron chelation — the trapping of harmful, unbound iron — is a possible therapy for FA. A 2008 study in
Frataxin production may be stimulated through the use of erythropoietin, a naturally occurring protein. In 2010, an Austrian research team
In June 2011, Edison Pharmaceuticals announced preliminary results from a 28-day phase 2a clinical trial of its experimental compound EPI-A0001
Antioxidants are designed to offer some protection against cell damage by chemicals known as free radicals and to enhance the
In the 1990s, the identification of mutations in the frataxin gene (from which frataxin protein is produced) as the underlying