Hospitalized infants with the rare metabolic disorder maple syrup urine disease (MSUD), who are intolerant to oral or enteral administration of branched-chain amino acid-free formula, may benefit from ...
Only transplant center that developed comprehensive protocol for liver transplants in kids with MSUD
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 transplants ...
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 ...
A study led by UMass Chan Medical School researchers has demonstrated that a gene therapy to correct a mutation that causes ...
The Asian Parent on MSN
Get to Know Maple Syrup Urine Disease, a Genetic Disease That Causes Sweet-Smelling Urine
In the first few weeks of life, babies are sometimes offered a newborn blood spot screening to check for an inherited condition such as maple syrup urine disease (MSUD). This is a very rare genetic ...
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 ...
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 ...
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