Researchers used AI to Repurpose Existing Drugs for Treatment of Rare Diseases

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On Sept. 25, 2024, a study led by scientists at Harvard Medical School announced they had developed an AI model, called TxGNN, that identified possible therapies from existing medicines for thousands of diseases, including rare ones with no current treatments. There are more than 7,000 rare and undiagnosed diseases globally that affect some 300 million people worldwide.

TxGNN identified drug candidates from existing medicines for more than 17,000 diseases, many of them without any existing treatments. This represented the largest number of diseases that any single AI model can handle to date.

The study was published in Nature Medicine. The researchers have made the tool available for free to encourage clinician-scientists to use it in their search for new therapies, especially for conditions with no or with limited treatment options.

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Source: Harvard University
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