PTC wins FDA approval for first brain-delivered gene therapy Kebilidi
Fierce Pharma
NOVEMBER 14, 2024
The FDA issued its first stamp of approval for a cell or gene therapy back in 2017 to Novartis' Kymriah. | The gene therapy has already been approved to treat AADC deficiency in Europe and the U.K., among other countries, where it's branded as Upstaza.
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