Relief Therapeutics and Acer Therapeutics Announce that the European Commission Has Granted Orphan Drug Designation for ACER-001 in Maple Syrup Urine Disease 12-Aug-2022 / 07:00 CET/CEST Release of an ...
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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 ...
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 ...
Novel BCKDHB variant and 6q14.1 deletion tied to double diagnosis of maple syrup urine disease and CHUJANS March 22, 2023 ...
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 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 ...
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