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 ...
The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has granted an exceptional circumstances positive opinion for Maapliv, a solution of amino acids ...
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 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 ...
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 ...
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 ...
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