Highlights:
Rett Syndrome program - 16 patients enrolled at Texas Children's Hospital, 7 of whom have completed the entire study. The study medicine appears to be well tolerated in this population. No serious adverse events (SAEs) have been reported and no subjects have discontinued treatment due to side effects.
A second site, the University of Alabama at Birmingham, will be added in August to further ensure that the enrolment target is met. The study is on track to complete enrolment in the first half of 2014 with top-line results in the second half of 2014.
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