aromatic L-amino acid decarboxylase (AADC); dopa-decarboxylase gene therapy; eladocagene exuparvovec (Kebilidi)
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Indications
- treatment of AADC deficiency (FDA-approved Nov 2024)[3]
- treatment of patients with moderately advanced Parkinson's disease due to AADC deficiency
Clinical significance
- AADC is deficient in putamen of patient with Parkinson's disease
- AADC converts convert levodopa to dopamine
Procedure
- adenovirus vector serotype-2 carrying cDNA encoding AADC
- coadministered bilaterally with gadoteridol into the putamen
- intraoperative MRI was used to visualize spread of the infusion
Clinical trials
- PD-1101 phase 1b trial
- well-tolerated & resulted in stable or improved motor function & quality of life across cohorts, & reduced PD medication requirements over 3 years[2]
Additional terms
References
- ↑ Christine CW, Bankiewicz KS, Van Laar AD et al. Magnetic resonance imaging-guided phase 1 trial of putaminal AADC gene therapy for Parkinson's disease. Ann Neurol. 2019 May;85(5):704-714. Epub 2019 Mar 26. PMID: https://www.ncbi.nlm.nih.gov/pubmed/30802998 https://onlinelibrary.wiley.com/doi/full/10.1002/ana.25450
- ↑ 2.0 2.1 Christine CW, Richardson RM, Van Laar AD et al. Safety of AADC gene therapy for moderately advanced Parkinson disease: Three-year outcomes from the PD-1101 trial. Neurology 2021 Oct 14; [e-pub]. PMID: https://www.ncbi.nlm.nih.gov/pubmed/34649873 https://n.neurology.org/content/early/2021/10/14/WNL.0000000000012952
- ↑ 3.0 3.1 George J First Brain-Delivered Gene Therapy Approved for AADC Deficiency. Novel treatment is indicated for children and adults with the rare disorder. MedPage Today November 14, 2024 https://www.medpagetoday.com/neurology/generalneurology/112915