An ultra-rare enzyme deficiency that keeps infants from achieving proper muscle function among other developmental delays now has its first FDA-approved treatment. The new product, a gene therapy ...
On Thursday, the FDA granted accelerated approval to PTC Therapeutics, Inc.’s (NASDAQ:PTCT) gene therapy for AADC deficiency, the first-ever gene therapy approved in the U.S. that is directly ...
WARREN, N.J., Nov. 13, 2024 /PRNewswire/ -- PTC Therapeutics (PTCT), Inc. announced today the U.S. Food and Drug Administration (FDA) accelerated approval of its gene therapy for the treatment of AADC ...
When the Human Genome Project concluded 21 years ago, it opened the door for genetic testing and a promise for lifesaving screenings and personalized medicine. An innovation that serves as a key ...
Some results have been hidden because they may be inaccessible to you
Show inaccessible results
Feedback