Voices of Friedreich's Ataxia (FA)

When pharmaceutical companies begin the journey of developing a drug for F A, one of the first questions asked is

Patients can accelerate the drug development and treatment process by registering for Friedreich’s Ataxia Global Patient Registry. Being part of

F A is known as a triplet repeat disorder since it is caused by an expanded trinucleotide repeat. In those

While no treatment is currently approved to halt the progression of the disease, there are more than a dozen therapies

A clinical trial evaluates a specific intervention according to a research protocol created by investigators. These interventions may be products

Frataxin, the protein produced by the FXN gene, works in mitochondria to help to create energy. Normally, frataxin works to

Genetic testing analyzes the FXN gene, which is the only gene identified to cause Friedreich’s ataxia. Positive genetic test results

F A is an inherited or single-gene disorder. Genetic Mutations or DNA changes in the FXN gene cause F A.

Many physicians and individuals with F A consider vitamin and supplement regimens. Clinical studies have suggested modest or subjective benefits

Genetic testing is diagnostic in more than 98% of individuals. In more than 95% of abnormal alleles, the mutation is