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Member Company BioMarin Develops Treatment for Rare Disease

Submitted on: 05.07.2008

CHI member company BioMarin (Novato, Calif.) has recently created a treatment called Kuvan  for the rare disease phenylketonuria or PKU.   PKU affects the liver where a patient is missing an enzyme to properly manage proteins in the body.  Patients with this rare disease must follow a strict low protein diet.  If a strict diet is not followed the devastating disease can cause brain damage.  The Kuvan drug treatment, recently approved by the  U.S.  Food and Drug Administration (FDA), works by activating  an  enzyme in a patient's body that helps manage phenylalanine levels.

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