Columnist Joe MacDonald reflects on the time he and his son spent building with Legos in the hospital as a distraction from hemophilia.

Experts explored the latest trends, approvals, and pipeline developments in cell and gene therapies, rare disease, immunology ...

Only a small group of patients have started the process to receive the new gene therapies for sickle cell disease, says Kevin ...

The U.S. Food and Drug Administration (FDA) has approved Hympavzi (marstacimab-hncq) for routine prophylaxis aimed at ...

The U.S. Food and Drug Administration (FDA) approved Hympavzi (marstacimab-hncq) for routine prophylaxis to prevent or reduce ...

Half of people who develop blood poisoning, otherwise known as sepsis, are dead within a couple of years, a new study finds.

Spanish authorities issued a positive recommendation for national reimbursement of Hemgenix gene therapy for eligible ...

The Food and Drug Administration (FDA) has approved Hympavzi (marstacimab-hncq) for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients 12 years ...

Although JT was born with Hemophilia A, a chronic bleeding disease, he was never one to be defined by it. JT lived a long ...

The FDA has approved a new preventive treatment for people with certain types of hemophilia that can reduce bleeding episodes, sometimes down to just a few per year.

A local pharmacist is bringing together federal and state leaders to explain why drug prices are so high and what needs to be done to change that.

The approval of Hympavzi Friday was a dose of good news for Pfizer, which is dealing with research setbacks and a challenge from an activist investor.