Last month, a baby got the world’s first personalized gene-editing treatment. What will this mean for millions of others with ...

Pfizer's hemophilia therapy, Hympavzi, met the main goal of a late-stage study in patients with certain types of antibodies, ...

For patients with recessive dystrophic epidermolysis bullosa (RDEB), a one-time surgical application of prademagene ...

In one of the first major tests of the new FDA leadership’s regulatory philosophy toward gene therapies for rare diseases, ...

The FDA is investigating two deaths in non-ambulatory Duchenne muscular dystrophy patients who received a Sarepta gene ...

In May 2025, researchers announced that K.J. Muldoon, a baby boy born without the ability to process dietary protein properly ...

The U.S. Food and Drug Administration said on Tuesday that it is investigating reports of two deaths due to acute liver ...