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Tag: Aadc deficiency

Explore our medication guides and pharmacology articles within this category.

Kebilidi: What was the first brain injected gene therapy approved by the FDA?

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4 min read
On November 14, 2024, the FDA granted accelerated approval to eladocagene exuparvovec-tneq (brand name Kebilidi), which was the first brain injected gene therapy approved by the FDA. This milestone marks a new era for treating severe, rare neurological disorders by directly targeting the source of the problem in the central nervous system.

Is kebilidi the same as upstaza?

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2 min read
The U.S. Food and Drug Administration (FDA) approved Kebilidi in November 2024, approximately two years after the same gene therapy was approved in the European Union (EU) as Upstaza. This means that when asking, 'Is kebilidi the same as upstaza?', the answer is yes: they are different brand names for the exact same medical product, `eladocagene exuparvovec`.