AI Model Finds Drug Candidates for Rare Diseases

Harvard Medical School

There are more than 7,000 rare and undiagnosed diseases globally.

  • By EKATERINA PESHEVA

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.

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