Casgevy, now a "national priority" drug, helped kids with sickle cell and thalassemia, results which may help toward a label ...

To the research team working to save him, KJ Muldoon was first known only as Patient Eta. But within months, KJ’s name — and ...

A rare disease mom and scientist shares on the "First Opinion Podcast" how she serves as a translator between her two ...

Lucy Landman was born with a very rare genetic disorder that causes severe intellectual disability, weak muscles and seizures, among other symptoms. "She is expected to very much never be able to live ...

A cutting-edge base-edited CAR T-cell therapy is opening a revolutionary new frontier for patients battling aggressive T-cell ...

The gene-editing technique is effective for treating some illnesses but it's been too expensive to consider it for rare conditions. A new approach... Lucy Landman was born with a very rare genetic ...