Latest Inborn errors of metabolism Stories
CRANBURY, N.J., Aug.
Hyperion Therapeutics has completed patient enrollment in its Phase I/II clinical trial to evaluate the safety, tolerability and ammonia scavenging effects of HPN-100 versus Buphenyl in patients with urea cycle disorders. The company plans to announce top-line results in the fourth quarter of 2008.
Asubio Pharma, one of the group companies of Daiichi Sankyo, has announced an additional indication for the inherited metabolic disease agent Biopten for the treatment of tetrahydrobiopterin responsive hyperphenylalaninemia.
NOVATO, Calif., July 16 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq and SWX: BMRN) announced today that Asubio Pharma Co., Ltd.
A gene therapy treatment that restores a missing liver enzyme in test animals could provide a cure for a rare metabolic disorder in humans, according to Duke University Medical Center researchers.
Given that BioMarin Pharmaceuticals's Kuvan is potentially the only treatment for the rare genetic metabolic disorder phenylketonuria, Datamonitor believes that Wall Street may be underestimating Kuvan's market potential.
Many parents have visited the NetWellness Web site with questions about their child's body odor. Luckily, body odor in itself usually does not signify a problem. As children mature, it is not uncommon to begin to have issues with body odor.