FDA approval: Kebilidi (from Catapult's regulatory round-up for November 2024)

FDA has approvedeladocagene exuparvovec-tneq (Kebilidi) a recombinant adeno-associated virus serotype 2 (rAAV2)-based gene therapy, containing the human DDC gene (dopa decarboxylase) indicated for the treatment of adult and paediatric patients with aromatic L-amino acid decarboxylase (AADC) deficiency. It is designed to correct the underlying genetic defect by delivering a functioning DDC gene directly into the putamen, increasing the AADC enzyme and restoring dopamine production. AADC defi...

Publication Details

Journal
Industrias Culturais (Universidade de Coimbra)
Published
2025-02-12
DOI
https://doi.org/10.58079/13axk
Primary Topic
Hydraulic flow and structures
Type
article
Field-Weighted Citation Impact
0.00
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article

FDA approval: Kebilidi (from Catapult's regulatory round-up for November 2024)

Industrias Culturais (Universidade de Coimbra)
Hydraulic flow and structures
article

FDA approval: Kebilidi (from Catapult's regulatory round-up for November 2024)

article en

Abstract

FDA has approvedeladocagene exuparvovec-tneq (Kebilidi) a recombinant adeno-associated virus serotype 2 (rAAV2)-based gene therapy, containing the human DDC gene (dopa decarboxylase) indicated for the treatment of adult and paediatric patients with aromatic L-amino acid decarboxylase (AADC) deficiency. It is designed to correct the underlying genetic defect by delivering a functioning DDC gene directly into the putamen, increasing the AADC enzyme and restoring dopamine production. AADC defi...

Industrias Culturais (Universidade de Coimbra)
Good health and well-being
Openalex Percentile: Top 57%
Hydraulic flow and structures
DOIOpen Access PDFOpenAlex
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FDA approval: Kebilidi (from Catapult's regulatory round-up for November 2024) · Industrias Culturais (Universidade de Coimbra) (2025) | TGRS Research Map | TGRS