Sriansh Ojha was fully into his "terrible twos" on an April afternoon at Cincinnati Children's as he squealed and chased his ...

The U.S. Food and Drug Administration approved Kebilidi (eladocagene exuparvovec-tneq), an adeno-associated virus ...

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., ...

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