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They mapped all the cells critical for skull formation and examined how genetic mutations may cause soft spots in a newborn baby’s skull to fuse too early, constraining the developing brain’s ...

Now, seven years and many approvals later, innovation in the space has hit new heights with PTC Therapeutics’ Kebilidi, the first U.S. gene therapy that’s administered directly in the brain.

On Wednesday, PTC 19, formerly known as Invest 99L, was over 400 miles east of Isla Guanaja, Honduras, and 290 miles east-northeast of Cabo Gracias A Dios, near the Honduras-Nicaragua border ...

The FDA has followed in the footsteps of its European counterparts and granted accelerated approval to PTC Therapeutics’ gene therapy Kebilidi for AADC deficiency. It is the first approved gene ...

Credit: Gatot Adri via Shutterstock. The US Food and Drug Administration (FDA) has granted accelerated approval for PTC Therapeutics’ KEBILIDI, the first gene therapy in the US that is directly ...

WARREN, N.J., Nov. 13, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today the U.S. Food and Drug Administration (FDA) accelerated approval of its gene therapy for the ...