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!