The U.S. Food and Drug Administration (FDA) on Friday announced that it has approved Casgevy, the first CRISPR gene-editing therapy for sickle cell disease, paving the way for thousands of patients in ...

Doctors say new gene-based treatments could offer long-term relief — and possible cures — for people living with sickle cell disease. Sickle cell disease has long shortened lives in the United States, ...

Fifteen-year-old Jayden Wilsey of Falmouth is the first person at Boston Children's Hospital to receive human gene therapy for sickle cell disease, following approval of the therapy in 2024 by the U.S ...

Scientists have long known what causes sickle-cell disease and its devastating effects: a single mutation in one errant gene. But for decades, there has been only modest progress against an inherited ...

At age 45, Dr. Lakiea Bailey said, for the longest time, that she was the oldest person with sickle cell anemia that she knew. The executive director of the nonprofit patient advocacy group the Sickle ...

When Dr. Ambroise Wonkam walked into a panel on medical genetics out of curiosity, he had no idea it would shape the course of his career. Born in Cameroon, Wonkam has dedicated his career to studying ...

At the weekend in Kampala, a piece of news landed that could reshape the future of one of the world’s most neglected diseases. The United States Patent and Trademark Office (USPTO) had accepted a ...

Two Pee Dee siblings are officially cured of sickle cell disease after more than a decade of living with the genetic blood ...