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 ...
Casgevy, now a "national priority" drug, helped kids with sickle cell and thalassemia, results which may help toward a label ...
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 ...
To compete at iGEM, a sort of science Olympics, teens at a Georgia high school set their sights on finding a better way to ...
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 ...