Kebilidi is the first gene therapy approved by the FDA for administration directly into the brain, given via a cannula during a surgical procedure, and replaces the human dopa decarboxylase ...

It markets Upstaza, a gene therapy with the brand name Kebilidi in the United States. PTC Therapeutics, Inc. was incorporated in 1998 and is headquartered in Warren, New Jersey.

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

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, ...

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

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

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

The company has license and collaboration agreement with Novartis Pharmaceuticals Corporation to develop PTC518 Huntington's disease program. It markets Upstaza, a gene therapy with the brand name ...