Monday, October 14, 2013 9:11:46 AM
Overall, oral GCD was found to be safe and well tolerated in all 12 patients across all of the three doses tested. There were no serious adverse reactions reported and no patient discontinued the study prematurely. Presence of an enzyme was detected in patients' blood circulation and the enzyme demonstrated biological activity. In addition, some of the patients who suffered from thrombocytopenia and had low platelet counts demonstrated a meaningful improvement in platelet count. Accordingly, the trial has been extended to enroll and evaluate additional Gaucher patients with low platelet counts. The amended study is expected to conclude during the fourth quarter of 2013. Additional data from the phase I study will be presented at the 10th Annual WORLD Symposium, Feb 10-13, 2014 in San Diego, CA.
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