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 metabolic disorder that some babies are born with. It is caused by a defect in an enzyme that breaks down certain amino aci ...

Gene therapy targeting maple syrup urine disease (MSUD) has shown potential in preventing newborn death, normalizing growth, and stabilizing biomarkers in animal models, including calves and mice.