Scientists at the Sanford Burnham Prebys Medical Discovery Institute in California may have found a cure for Alagille Syndrome, a potentially fatal genetic disease that affects multiple organs, most notably the liver.. There are no known treatments for this disease..

Newly developed drug NoRA1 targets the Notch pathway, a cellular-level signaling system in the body that is important for many biological systems, including proper liver function, Science Alert reports..

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The genetic mutation that leads to the development of Alagille syndrome causes decreased Notch signaling. This, in turn, leads to impaired growth and regeneration of the bile ducts, causing bile accumulation and liver damage..

Studies in zebrafish have shown that NoRA1 enhances Notch signaling in these fish with the same genetic mutations as in children diagnosed with the disease.. As a result, the growth and regeneration of duct cells, as well as the regeneration of liver tissues, were launched, which made it possible to reverse damage to this organ in fish..

The treatment increased the chances of survival of fish diagnosed with the disease.. Scientists note that one of the advantages of their drug is that it is aimed at activating processes that should occur naturally..

It will be a plus if the drug can pass laboratory tests and be approved for human trials..

Agille syndrome is diagnosed in about four thousand newborns a year, often requiring a liver transplant. If this is not done quickly, the disease leads to death in 75% of cases..

Important! This publication is based on the latest and current scientific research in the field of medicine and is for general information purposes only.. Publication cannot be the basis for making any diagnoses. If you are ill or need a diagnosis, see your doctor!