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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 ...

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 ...

And while there are serums out there with *many* different vitamin C derivatives, I find that L-ascorbic acid serums give me ...

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

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Barclays raised the firm’s price target on PTC Therapeutics (PTCT) to $45 from $43 and keeps an Equal Weight rating on the shares. After ...

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 drug is a treatment for a rare and fatal genetic disorder called aromatic l-amino acid decarboxylase (AADC) deficiency, ...