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., ...

Gene therapy can effectively treat various diseases, but for some debilitating conditions like muscular dystrophies there is a big problem: size.

Gene therapy can effectively treat various diseases, but for some debilitating conditions like muscular dystrophies there is a big problem: size. The ...

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 U.S. Food and Drug Administration approved Kebilidi (eladocagene exuparvovec-tneq), an adeno-associated virus vector-based gene therapy indicated for the treatment of adult and pediatric patients ...

Ascend Advanced Therapies, which has been on an expansion tear since debuting three years ago, inked a deal with EW ...

KEBILIDI is authorized to be administered using the SmartFlow Neuro Cannula. KEBILIDI is an adeno-associated virus vector-based gene therapy indicated for the treatment of adult and pediatric patients ...

WARREN, NJ, USA I November 13, 2024 I PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today the U.S. Food and Drug Administration (FDA) accelerated approval of its gene therapy for the treatment of ...

Developed by PTC Therapeutics, Kebilidi is an adeno-associated virus vector-based gene therapy indicated for the treatment of ...

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

High dose TSHA-102 was generally well tolerated with no SAEs or DLTs in two adolescent/adult patients and one pediatric patient as of data ...