Although each condition occurs in a small number of individuals, collectively these diseases exert a staggering human and economic toll because they affect some 300 million people worldwide. Yet, with a mere 5 to 7 percent of these conditions having an FDA-approved drug, they remain largely untreated or undertreated.
Developing new medicines represents a daunting challenge, but a new artificial intelligence tool can propel the discovery of new therapies from existing medicines, offering hope for patients with rare and neglected conditions and for the clinicians who treat them.
The AI model, called TxGNN, is the first one developed specifically to identify drug candidates for rare diseases and conditions with no treatments.
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