PTC Therapeutics’ gene therapy, Kebilidi to treat AADC deficiency receives US FDA approval: Warren, New Jersey Friday, November 15, 2024, 10:00 Hrs [IST] PTC Therapeutics, Inc., ...

To determine the type and severity of a cancer, pathologists typically analyze thin slices of a tumor biopsy under a microscope.

StitchR, a new gene therapy technique, delivers large genes in two parts to treat muscular dystrophies by restoring critical proteins in animal models.

Kebilidi is the first approved gene therapy that can be directly administered to the brain. Its OK secures a priority review ...

The Food and Drug Administration (FDA) has granted accelerated approval to Kebilidi™ (eladocagene exuparvovec-tneq) for the treatment of adult and pediatric patients with aromatic L-amino acid ...

Gene therapy can effectively treat various diseases, but for some debilitating conditions like muscular dystrophies there is ...

By exploiting the genetic variation in cancer cells, an already approved cancer drug demonstrated enhanced effects against cancer cells in specific patient groups, according to a new study. The ...

PTC Therapeutics' Kebilidi gains FDA accelerated approval as the first brain-administered gene therapy in the U.S. for AADC ...

The recent FDA approval of Kebilidi, a groundbreaking gene therapy for AADC deficiency, is a significant milestone for the company. It is the first gene therapy directly administered to the brain and ...

This is the first gene therapy for AADC deficiency to receive US regulatory approval and the first that can be administered directly to the brain.

The therapy, developed by PTC Therapeutics, is designed for patients across the severity of spectrum of AADC deficiency. It ...