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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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Oman to tackle 9.5% genetic disease rate with enhanced newborn and premarital screening
29 Jan 2025 09:10 GMT
… conditions like phenylketonuria (PKU) and maple syrup urine disease (MSUD), as well as hormonal …
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Why Early Diagnosis Can Be a Lifesaver: The Role of Genetics in Rare Diseases?
28 Jan 2025 09:14 GMT
… which confirm the IEMs like Maple Syrup Urine Disease (MSUD), Urea Cycle defects (UCD …
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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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Strasburg clinic looking to cure patients with maple syrup urine disease
21 Apr 2023 21:20 GMT
… rare genetic disorders, such as maple syrup urine disease (MSUD).
According to the Children …
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What to know about maple syrup urine disease
07 Oct 2022 16:42 GMT
Maple syrup urine disease (MSUD) is a rare inherited …
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Woman who suffers from rare 'maple syrup urine disease' says that she may DIE over formula shortage
26 May 2022 20:40 GMT
… suffers from the rare 'maple syrup urine disease' says that the formula …
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Maple Syrup Urine Disease: Why The Formula Shortage Could Also Be Deadly For Some Adults
26 May 2022 00:34 GMT
… potentially deadly: people with maple syrup urine disease.
People with maple syrup urine disease can't break … in short supply.
What is maple syrup urine disease?
Maple syrup urine disease is a rare genetic disorder …
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I'm a 28-year-old woman and have maple syrup urine disease. The formula shortage could kill me.
24 May 2022 23:16 GMT
Hannah Dolins has maple syrup urine disease and needs a metabolic formula … a rare genetic disease called maple syrup urine disease, or MSUD. Only about 2 …