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Vitamin Treatment For Hereditary Ataxia Brings Dramatic Improvements

Date:
April 11, 2001
Source:
American Academy Of Neurology
Summary:
Researchers have discovered a new treatment for one form of the rare disorder hereditary ataxia that has resulted in remarkable improvements, according to a study in the April 10 issue of Neurology, the scientific journal of the American Academy of Neurology.

ST. PAUL, MN -- Researchers have discovered a new treatment for one form of the rare disorder hereditary ataxia that has resulted in remarkable improvements, according to a study in the April 10 issue of Neurology, the scientific journal of the American Academy of Neurology.

"An 8-year-old boy who was confined to a wheelchair was able to walk independently after the treatment, and a 20-year-old woman was able to work outside the home for the first time," said study author and neurologist Salvatore DiMauro, MD, of Columbia University in New York, NY.

Hereditary ataxia is a genetic neurological disorder that affects coordination. Patients have difficulty with balance, coordination of arms and legs and speech. Some patients also develop seizures. The disease often causes deterioration of the cerebellum, the area of the brain that controls coordination.

The researchers discovered that some patients with hereditary ataxia have a decreased level of coenzyme Q10, or CoQ10, in their muscles. CoQ10, also called ubiquinone, is a vitamin-like substance that plays a key role in the production of energy within cells. It is naturally present in small amounts in various foods.

For the study, the researchers identified people with hereditary ataxia with no known genetic cause. CoQ10 levels were low among the six patients identified -- about 70 percent lower than normal. The patients were then given daily supplements of CoQ10, ranging from 300 mg to 3,000 mg.

"All of the patients improved with the CoQ10," DiMauro said. "They got stronger, their ataxia improved and their seizures either stopped or happened less often.

One year after they started taking CoQ10, the patients' scores improved by an average of 25 percent on an ataxia scale measuring their balance, speech and movement. Five of the patients were unable to walk before receiving CoQ10; after treatment all were able to walk with some assistance, such as a rolling walker.

There are many forms of hereditary ataxia, also called hereditary spinocerebellar ataxia, or SCA. These patients did not have the autosomal dominant forms of SCA (SCA1 to SCA5) or Friedreich's ataxia.

"Our findings suggest that CoQ10 deficiency is a potentially important cause of some forms of familial ataxia and it should be considered when diagnosing this condition," DiMauro said. "Where low levels are found, treatment to replace the missing CoQ10 should be aggressive and begin early."

A neurologist is a medical doctor with specialized training in diagnosing, treating and managing disorders of the brain and nervous system.

The American Academy of Neurology, an association of more than 17,000 neurologists and neuroscience professionals, is dedicated to improving patient care through education and research.

For more information about the American Academy of Neurology, visit its web site at http://www.aan.com.


Story Source:

The above story is based on materials provided by American Academy Of Neurology. Note: Materials may be edited for content and length.


Cite This Page:

American Academy Of Neurology. "Vitamin Treatment For Hereditary Ataxia Brings Dramatic Improvements." ScienceDaily. ScienceDaily, 11 April 2001. <www.sciencedaily.com/releases/2001/04/010411080944.htm>.
American Academy Of Neurology. (2001, April 11). Vitamin Treatment For Hereditary Ataxia Brings Dramatic Improvements. ScienceDaily. Retrieved August 20, 2014 from www.sciencedaily.com/releases/2001/04/010411080944.htm
American Academy Of Neurology. "Vitamin Treatment For Hereditary Ataxia Brings Dramatic Improvements." ScienceDaily. www.sciencedaily.com/releases/2001/04/010411080944.htm (accessed August 20, 2014).

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