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 ...

A study led by UMass Chan researchers demonstrated that a gene therapy to correct a mutation that causes maple syrup urine disease (MSUD) prevented newborn death, normalized growth, restored ...

Maple syrup urine disease (MSUD) is a rare genetic inborn error of metabolism characterized by recurrent life-threatening neurologic crises and progressive brain ...

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A study in human skin cells and mice shows the widely-used diabetes drug metformin reduces toxic acid levels in Maple Syrup Urine Disease. The discovery offers the possibility of a new treatment for a ...

A study led by UMass Chan Medical School researchers has demonstrated that a gene therapy to correct a mutation that causes maple syrup urine disease (MSUD) prevented newborn death, normalized growth, ...

A new study analyzes 30 years of patient data and details the clinical course of 184 individuals with genetically diverse forms of Maple Syrup Urine Disease (MSUD), which is among the most volatile ...

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 ...

Hannah Dolins has maple syrup urine disease and needs a metabolic formula to live. Without her formula, she can fall into a coma and die. This is her story, as told to Heather Marcoux. This as-told-to ...

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 ...