NOVATO, Calif., Jan. 11 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. announced today that the first subject has initiated treatment in the Phase 1 clinical study of BMN 195, a small molecule utrophin upregulator, for the treatment of Duchenne muscular dystrohpy (DMD). Initial top-line results are expected in the third quarter of 2010.
The Phase 1 clinical trial is a single-center, double-blind, placebo-controlled, single-dose escalation study followed by a multiple-dose escalation study of BMN 195 administered orally in healthy volunteers. The primary objective is to assess the safety, tolerability and pharmacokinetics of BMN 195 in healthy volunteers, and enable subsequent studies in patients with DMD.
Duchenne muscular dystrophy is a fatal neuromuscular disorder that affects 1 in 3,500 boys with an estimated patient population of over 40,000 in the developed world.
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