Drug for rare disease effective for alzheimers, autism treatment -study

Drug for rare disease effective for alzheimers, autism treatment -study

The drug, meant to treat symptoms related to ADNP syndrome – a rare genetic disease that causes symptoms such as intellectual disability and autism spectrum disorder – has other applications. An RNA drug is seen being delivered, targeting infected cells (Illustrative). (photo credit: TEL AVIV UNIVERSITY) A Tel Aviv University study found that a drug that was designated as an “ orphan drug ” by the FDA – meaning it is used to treat rare diseases and is subject to separate regulations – can also treat a variety of symptoms associated with autism, intellectual disabilities, and Alzheimer’s disease. Prof. Illana Gozes of the Tel Aviv University Sackler Medical School’s Department of Human Molecular Genetics and Biochemistry discovered the drug – named NAP – in 2020. The drug is meant to treat symptoms related to ADNP syndrome, a rare genetic disease that causes symptoms such as intellectual disability and autism spectrum disorder. Prof. Gozes then commissioned this most recent study , which was published in the peer-reviewed scientific journal Biological Psychiatry , with the intention of studying the drug’s effect on those suffering from diseases other than ADNP that also exhibit similar mental symptoms. “NAP, in fact, comprises a short […]

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