Scientists have used personalised gene-editing therapy to successfully treat a baby with a rare genetic condition. Could this ...

On Saturday, May 24, the 78th World Health Assembly (WHA) voted to adopt a resolution to establish a global framework to ...

An infant with a severe genetic condition has shown signs of improvement after receiving a gene-editing treatment tailored to ...

Jim Wilson‘s pioneering gene therapy explorations have helped launch eight companies focused on translating his 40 years of ...

Ocugen's drug to treat retina diseases received a rare pediatric disease designation from U.S. regulators. The biotechnology said Tuesday that the Food and Drug Administration granted the designation ...

Treatment seems to have been effective, but it is not clear whether such bespoke therapies can be widely applied.

An infant with a rare urea cycle disorder became the first patient to receive a personalized gene-editing therapy. His care ...

STATEN ISLAND, N.Y. — A Graniteville woman has been diagnosed with the rarest and deadliest brain disorder in the world, her ...

Family overjoyed correction to DNA defect that causes metabolic disorder has given KJ Muldoon a new lease of life ...

While sparking excitement among biopharma companies focused on rare and ultrarare indications, experts say FDA Commissioner ...

At the entrance of the 2025 International Conference on Rare Diseases, visitors are greeted by a poignant painting: a young ...