What Causes Maple Syrup Urine Disease? Maple syrup syndrome is a very rare genetic disorder, with only about 1 in 185,000 infants born with the condition. MSUD is caused by mutations or changes in ...
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 ...
Most days, you probably don’t pay much attention to what your urine smells like. But if you notice an unexpected odor, it’s hard not to take notice. Occasionally, some people notice their urine has a ...
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 ...
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) is a rare genetic inborn error of metabolism characterized by recurrent life-threatening neurologic crises and progressive brain ...
Some results have been hidden because they may be inaccessible to you
Show inaccessible results
Feedback