INDIANAPOLIS — With Rare Disease Day approaching, an Indiana family is bringing awareness to their daughter’s uncommon condition. FOX59 in Indianapolis recently told the story of a 4-month-old girl ...
One year ago, Children's Hospital of Pittsburgh became the first and only transplant center in the world to develop a comprehensive, multidisciplinary medical protocol for performing liver ...
Maple syrup urine disease (MSUD) is a rare genetic inborn error of metabolism characterized by recurrent life-threatening neurologic crises and progressive brain ...
A one-time injection of a novel gene therapy was able to save mice and a newborn calf from succumbing to maple syrup urine disease (MSUD), suggesting the approach could one day be used to treat ...
MILPITAS, Calif.--(BUSINESS WIRE)--ASC Therapeutics, a privately-held biopharmaceutical company pioneering the development of transformative in-vivo gene replacement, gene editing and allogeneic cell ...
MILPITAS, Calif.--(BUSINESS WIRE)--Please replace the release dated May 16, 2022 with the following corrected version due to multiple revisions. The updated release ...
Maple syrup urine disease (MSUD) is caused by an inherited deficiency of the branched-chain α-keto acid dehydrogenase, resulting in a marked increase of the branched-chain amino acids (BCAAs), leucine ...
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