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Genzyme Corp.
FDA approves first treatment
May 14th, 2003
The U.S. Food and Drug Administration (FDA) has approved the first treatment for patients with Fabry Disease, a serious metabolic genetic disorder affecting approximately 1 in 40,000 males. While it is believed that fewer females suffer the most serious consequences of the disease, they can be similarly and seriously affected as well. Because of a deficiency in an enzyme, alpha-galactosidase A, Fabry disease causes certain fats to accumulate in the blood vessels over many years, leading to the involvement of various tissues and organs of the body, including the kidneys and the heart, which can then cause organ failure. As a result, patients with Fabry disease often must...
Source: Biotech Week (2003-05-14)
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