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BioMarin Making Big Steps in Batten Disease

Posted by Bee For Battens in Research News | 0 comments

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BioMarin seeks to test enzyme-replacement therapy in Batten Disease patients


BioMarin Pharmaceutical Inc. doesn't back down from a tough rare disease.

The Novato company (NASDAQ: BMRN), which made its name with enzyme-replacement therapies that tackle virtually unpronounceable orphan diseases like phenylketonuria and versions of mucopolysaccharidosis, expects to enroll the first patient by mid-year in an early-stage clinical trial in Batten Disease.

CEO Jean-Jacques Bienaimé said BioMarin will file an investigational new drug application with the Food and Drug Administration -- the first step toward a human clinical trial -- yet this quarter.

Full Article Here!

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