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 ...

Animal model testing of the digenic gene therapy found it restored enzyme activity, improved growth, and prevented death.

Sweet-smelling urine can be an early sign of diabetes. Diabetic ketoacidosis is a medical emergency and requires immediate hospitalization. If you notice a maple syrup-like smell in your baby's urine, ...

Maple Syrup Urine Disease (MSUD) is a rare inherited metabolic disorder involving the dysfunction of an enzyme which breaks down three essential amino acids: leucine, isoleucine and valine. Left ...

Researchers from the University of Pennsylvania, Perelman School of Medicine, Gene Therapy Program, and Moderna, have shown that repeated administration of lipid nanoparticle-encapsulated mRNA therapy ...

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 groundbreaking study by researchers at the University of Pennsylvania and Moderna has shown that repeated mRNA therapy can significantly improve survival and reduce leucine levels in a mouse model ...

New Rochelle, NY, August 21, 2024—Researchers from the University of Pennsylvania, Perelman School of Medicine, Gene Therapy Program, and Moderna, have shown that repeated administration of lipid ...

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