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Highlights from BioMarin's Research & Development Day
Date:12/8/2011

NOVATO, Calif., Dec. 8, 2011 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today hosted a Research and Development Day where members of the company's management team and industry experts provided an update on BioMarin's product portfolio and advancements in the research and development pipeline.

"We believe 2011 has been a year of significant progress in both our late stage and early stage clinical development programs," said Jean-Jacques Bienaime, Chief Executive Officer of BioMarin.  "This progress sets the stage for multiple data readouts in the coming year from key programs throughout our pipeline.  We believe these events will help move us forward in delivering more therapies that could make large impacts on the lives of patients suffering from several rare diseases."

Program Highlights:GALNS for MPS IVA

  • Phase I/II extension data shows continued efficacy
  • Endurance benefits in 6 minute walk test, 3 minute stair climb and pulmonary function tests are sustained
  • Urinary keratan sulfate (uKS) drop is sustained
  • Preliminary growth data encouraging, and biochemical markers suggests that GALNS reaches the cartilages and  improves chondrocyte activity
  • Safety data consistent with earlier results
  • Phase 3 study on track: Enrollment and site quality
  • Risk mitigation strategy implemented
  • BMN-701 for Pompe Disease

  • Good safety profile to date in Phase 1/2 study and cleared to start 20 mg/kg cohort
  • Positive efficacy trends in 5 mg/kg cohort (three patients)
  • Reduced substrate (Urinary tetrasaccharide) in two patients
  • Large improvement in 6 minute walk test in one patient
  • Improved respiratory function in three patients
  • On track to report Phase 1/2 results in 2H 2012
  • PEG-PAL for PKU'/>"/>

    SOURCE BioMarin Pharmaceutical Inc.
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