FDA Approves Kuvan For Phenylketonuria Treatment

Armen Hareyan's picture
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FDA approved Kuvan (sapropterin dihydrochloride), the first drug of its kind approved to slow the effects of a rare genetic disorder that causes mental retardation, smaller brain size, delayed speech and other neurological problems. Tetrahydrobiopterin (BH4)-responsive phenylketonuria or PKU disease occurs in one out of every 12,000 to 15,000 live births in the United States.

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