Ataxia with oculomotor apraxia
Ataxia with oculomotor apraxia is a group of rare genetic disorders characterized by progressive loss of coordination and difficulty with side-to-side eye movements.
Overview
Ataxia with oculomotor apraxia is a condition characterized by movement problems that worsen over time. The hallmark symptom is ataxia, or poor coordination and balance. Most affected individuals also have oculomotor apraxia, which makes it difficult to move the eyes side-to-side, requiring the person to turn their head to see objects in their peripheral vision.
There are several types of this condition, with types 1, 2, and 4 being the most common. While the types are similar, they are caused by mutations in different genes.
Types and Symptoms
- Type 1: Begins around age 4. Symptoms include ataxia, oculomotor apraxia, involuntary jerking movements (chorea), or muscle twitches (myoclonus). Individuals may develop muscle wasting in the hands and feet and neuropathy, which impairs reflexes and causes limb weakness.
- Type 2: Usually begins around age 15. Similar to type 1, it involves ataxia, oculomotor apraxia, chorea, myoclonus, and neuropathy. A key feature is high levels of alpha-fetoprotein (AFP) in the blood.
- Type 4: Begins around age 4. In addition to ataxia and oculomotor apraxia, individuals typically develop dystonia (involuntary, sustained muscle tensing). Muscle wasting and neuropathy are also common.
Causes
Mutations in the APTX, SETX, or PNKP genes cause types 1, 2, and 4, respectively. These genes provide instructions for proteins involved in repairing damaged DNA. A shortage of these functional proteins leads to the accumulation of broken DNA strands, which can cause cell death. The cerebellum, the part of the brain involved in coordinating movements, is especially at risk, and the loss of brain cells there is thought to cause the characteristic movement problems.
Genes Associated with Ataxia with Oculomotor Apraxia
- APTX
- PNKP
- SETX
Inheritance
All types of this condition are inherited in an autosomal recessive pattern, meaning both copies of the gene in each cell have mutations. Parents of an affected individual each carry one copy of the mutated gene but typically do not show signs or symptoms.
Other Names for This Condition
- Adult onset ataxia with oculomotor apraxia
- EAOH
- Early-onset ataxia with ocular motor apraxia and hypoalbuminemia
- SCAN2
- SCAR1
- Spinocerebellar ataxia with axonal neuropathy type 2
- Spinocerebellar ataxia, recessive, non-Friedreich type 1