What is Friedreich's Ataxia?
Most individuals have onset of symptoms of FA between the ages of 5 and 18 years. Adult or late onset FA is less common, <25% of diagnosed individuals, and can occur anytime during adulthood. FARA is supporting research that will improve the quality and length of life for those diagnosed with Friedreich's ataxia and will lead to treatments that eliminate its symptoms.
The Signs & Symptoms of Friedreich's Ataxia are:
loss of coordination (ataxia) in the arms and legs
fatigue - energy deprivation and muscle loss
vision impairment, hearing loss, and slurred speech
aggressive scoliosis (curvature of the spine)
diabetes mellitus (insulin - dependent, in most cases)
serious heart conditions, including hypertrophic cardiomyopathy and arrhythmias
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These symptoms are not present in all individuals with FA, for example diabetes occurs in about 10-20% of individuals with FA. The mental capabilities of people with Friedreich's ataxia remain completely intact. The progressive loss of coordination and muscle strength leads to motor incapacitation and the full-time use of a wheelchair. Most young people diagnosed with FA require mobility aids such as a cane, walker, or wheelchair by their teens or early 20s.
Research pipeline
FARA is supporting the advancement of these treatments with financial resources, advocacy, patient engagement and/or fostered collaboration. FARA believes that there is merit in each of these approaches and that effective treatment will come in the form of a "cocktail approach" - a combination of two or more therapies.
This pipeline has been updated as of February 2019 and focuses on drugs that are already in preclinical development or have advanced to human clinical trial.
