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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 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 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 ...
STRASBURG, PA- 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 ...
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 ...
Novel BCKDHB variant and 6q14.1 deletion tied to double diagnosis of maple syrup urine disease and CHUJANS March 22, 2023 ...
Dreyfus, P. M., and Hauser, G., Microchimica Acta, II, 150 (1964). Dreyfus, P. M., and Hauser, G., Biochim. Biophys. Acta, 104, 78 (1965).
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