Phase I/II

SOURCE BioMarin Pharmaceutical Inc.


The GREAT news is out! I waited for the fundraiser to announce to everyone there the press release that was sent to us earlier in the month from Biomarin, the pharmaceutical company conducting the Phase I/II clinical trials on a potential treatment for Trey’s disorder, MPS IVA. God hear our prayers 

BIOMARIN REPORTS ENCOURAGING PRELIMINARY DATA ON BMN 110 FOR MPS IVA!!!

Their key observations were:
• Keratan sulfate levels fall within a few weeks after the start of therapy
• Improvements in 6-minute walk distance and 3-minute stair climb at 24 weeks are consistent with those observed with clinical studies for MPS I, MPS II and MPS IV (Trey's original diagnosis and treatment)
• The frequency and severity of infusion reactions appear comparable to those observed with Naglazyme (Trey's original infusion drug) and Aldurazyme.

“Although still early, we are encouraged by these initial signals of efficacy of GALNS enzyme replacement therapy for Morquio disease (MPS IVA). We plan to work closely with the FDA and other health authorities to finalize a Phase III protocol after the completion of the current study and have increased confidence that we will initiate a Phase III registration-enabling program by the fourth quarter of 2010 or the first quarter of 2011.”