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AAV Gene Therapy for Maple Syrup Urine Disease Shows Promise
04 Mar 2025 13:08 GMT
Maple syrup urine disease (MSUD) is a rare genetic … and a calf with classic maple syrup urine disease.”
“Simply put, we believe the …
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Study demonstrates successful gene therapy for maple syrup urine disease
02 Mar 2025 07:52 GMT
… correct a mutation that causes maple syrup urine disease (MSUD) prevented newborn death, normalized … and a calf with classic maple syrup urine disease. Science Translational Medicine. doi.org …
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Gene therapy for maple syrup urine disease shows promise
01 Mar 2025 16:30 GMT
… a debilitating genetic disorder called maple syrup urine disease (MSUD). The treatment can prevent …
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Gene Therapy for Maple Syrup Urine Disease Shows Promise in Animal Study
28 Feb 2025 17:42 GMT
… correct a mutation that causes maple syrup urine disease (MSUD) prevented newborn death, normalized … and a calf with classic maple syrup urine disease. Sci Transl Med. 17(787 …
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The Growing Landscape of the Amino Acid Metabolism Disorders Treatment Market
13 Mar 2025 12:12 GMT
… conditions such as phenylketonuria (PKU), maple syrup urine disease (MSUD), and homocystinuria significantly … 2031.
By Indication
• Phenylketonuria (PKU)
• Maple Syrup Urine Disease (MSUD)
• Citrullinemia
• Homocystinuria
• Argininosuccinic Aciduria …
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mRNA therapy shows promise for maple syrup urine disease
22 Aug 2024 04:58 GMT
… in a mouse model of maple syrup urine disease (MSUD).
The researchers, led by … Acids in Mouse Models of Maple Syrup Urine Disease. Human Gene Therapy. doi.org …
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Lipid nanoparticle mRNA therapy improves survival in mouse models of maple syrup urine disease
21 Aug 2024 20:52 GMT
… in a mouse model of maple syrup urine disease (MSUD).
The work appears in … Acids in Mouse Models of Maple Syrup Urine Disease, Human Gene Therapy (2024). DOI … survival in mouse models of maple syrup urine disease (2024, August 21)
retrieved 21 …
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Ava’s doctors hadn’t heard of maple syrup urine disease – but a new helpline for rare conditions could help those like her
17 Dec 2023 14:28 GMT
… people her daughter Ava has maple syrup urine disease, most people, not even medical …
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'My daughter was diagnosed with rare disease after I noticed sweet syrup smell on her'
28 Feb 2025 17:20 GMT
… #39;s unique condition - Maple Syrup Urine Disease (MSUD). MSUD is an … just for Maple Syrup Urine Disease, but for the rare disease community." Maple Syrup Urine Disease (MSUD … the tell-tale signs of Maple Syrup Urine Disease (MSUD) is sweet-smelling …
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Mum shares rare disease warning after spotting 'sweet syrup' smell on her daughter
28 Feb 2025 14:53 GMT
… 's unusual disorder - Maple Syrup Urine Disease (MSUD). MSUD is an rare … bigger things, not just for Maple Syrup Urine Disease, but for the rare disease … for various inherited conditions, including Maple Syrup Urine Disease (MSUD), around five days after …