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ALS

Amyotrophic Lateral Sclerosis (ALS, sometimes called Maladie de Charcot) is a progressive, usually fatal, neurodegenerative disease caused by the degeneration of motor neurons, the nerve cells in the central nervous system that control voluntary muscle movement. In the United States, the condition is often referred to as Lou Gehrig's Disease, after the New York Yankees baseball star who was diagnosed with the disease in 1939 and died from it two years later; today, renowned physicist Stephen Hawking is likely the best-known living ALS patient.

The disorder causes muscle weakness and atrophy throughout the body as both the upper and lower motor neurons degenerate, ceasing to send messages to muscles. Unable to function, the muscles gradually weaken, develop fasciculations (twitches) because of denervation, and eventually atrophy because of that denervation. The patient may ultimately lose the ability to initiate and control all voluntary movement; bladder and bowel sphincters and the muscles responsible for eye movement are usually (but not always) spared.

Cognitive function is generally spared except in certain situations such as when ALS is associated with frontotemporal dementia.[1] However, there are reports of more subtle cognitive changes of the frontotemporal type in many patients when detailed neuropsychological testing is employed. Sensory nerves and the autonomic nervous system, which controls functions like sweating, generally remain functional.

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Research for a Cure

The new discovery of RNAi has some promise in treating ALS. In recent studies, RNAi has been used in lab rats to shut off specific genes that lead to ALS. Cytrx Corporation has sponsored ALS research utilizing RNAi gene silencing technology targeted at the mutant SOD1 gene.

The mutant SOD1 gene is responsible for causing ALS in a subset of the 10% of all ALS patients who suffer from the familial, or genetic, form of the disease. Cytrx's orally-administered drug Arimoclomol is currently in clinical evaluation as a therapeutic treatment for ALS.

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Treatment

There is no cure for ALS, but treatment can help patients stay strong and independent for as long as possible. It can also help patients avoid other problems from ALS.

Physical and occupational therapy can help patients stay strong and make the most of the abilities patients still have. Speech therapy can help patients keep patients ability to talk after problems with speech begin. And there are several medicines that doctors can use to help relieve patients symptoms and keep patients comfortable.

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