Hyperion Therapeutics today announced that the first patient has been enrolled in a clinical trial of investigational compound HPN-100 (glycerol phenylbutyrate) in children ages 6-17 with urea cycle disorders (UCD). The study will evaluate the safety, tolerability, and ammonia control of glycerol phenylbutyrate compared to BUPHENYL® (sodium phenylbutyrate). The 2-3 week trial will enroll ten children currently treated with sodium phenylbutyrate for their UCD and will be followed by a one year open-label safety extension. The pivotal efficacy study in adult UCD patients will be conducted in parallel beginning later this year.
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