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Nov 12, 2012
 |  BC Week In Review  |  Clinical News  |  Clinical Results

GALNS: Phase III data

The double-blind, international Phase III MOR-004 trial in 176 patients with MPS-IVA showed that once-weekly 2 mg/kg IV GALNS met the primary endpoint of improving 6MWD from baseline to week 24 vs. placebo, while 2 mg/kg GALNS given every other week missed the endpoint. Specifically, once-weekly GALNS led to a mean increase of 22.5 meters in 6MWD over placebo (p=0.0174). Observed mean improvements in 6MWD from baseline to week 24 were 13.5 meters for placebo, 36.5 meters for once-weekly GALNS and 14.9 meters for GALNS...

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