| Autor: |
Hwu, P. W.-L., Lee, N.-C., Chien, Y.-H., Russell, A., Sierra, J. R., Wang, A., Grünert, J., Tai, C.-H. |
| Předmět: |
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| Zdroj: |
Neuropediatrics; 2023 Supplement 1, Vol. 54, pS1-S32, 32p |
| Abstrakt: |
This article, published in the journal Neuropediatrics, discusses the impact of gene therapy on cognitive and language development in children with aromatic L-amino acid decarboxylase (AADC) deficiency. AADC deficiency is a rare genetic disorder that affects the central nervous system and impairs cognitive and motor development. The study found that gene therapy with eladocagene exuparvovec resulted in gradual and sustained increases in cognitive and language scores, as measured by the Bayley-III scales. These findings suggest that gene therapy may be a promising treatment option for children with AADC deficiency. [Extracted from the article] |
| Databáze: |
Complementary Index |
| Externí odkaz: |
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