CenExel CNS is looking for participants with Friedreich Ataxia for a clinical trial. Friedreich Ataxia (FA) is a rare and inherited disease causing movement problems and progressive damage to the nervous system. Nerve fibers in the spinal cord and peripheral nerves degenerate, weaken, and become thinner. Peripheral nerves signal muscles to generate movement by carrying information back and forth between the brain and the body. To a lesser degree, the cerebellum (which coordinates balance and movement) also degenerates. Symptoms usually become noticeable between 5 and 15 years old, although they may appear after age 25.
Friedreich Ataxia does NOT affect thinking and reasoning abilities though – cognitive functions remain.
Symptoms may include:
- Unsteady balance and difficulty walking (gait ataxia)
- Awkward movements and impaired muscle coordination (ataxia), getting worse over time
- Loss of normal reflexes, especially in knees and ankles
- Impaired sensory functions
- Increased muscle tone (spasticity)
- Trouble swallowing (problems coordinating tongue and throat muscles)
- Hearing or vision loss
- Slowness or slurring of speech (dysarthria)
- Curving of the spine (scoliosis)
- Heart disease or diabetes
Friedreich Ataxia can shorten life expectancy, but some people with less severe cases live into their sixties or beyond.