Crigler-Najjar syndrome: looking to the future does not make us forget the present
Abstract Recently, the safety and efficacy of gene therapy were evaluated in patients with Crigler-Najjar syndrome (CNS). Although it is a promising curative option for CNS, many doubts still persist about its long-term efficacy and safety. Furthermore, there is a risk of overlooking several unresol...
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| 主要な著者: | , , |
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| フォーマット: | Artigo |
| 言語: | Inglês |
| 出版事項: |
BMC
2024-03-01
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| シリーズ: | Orphanet Journal of Rare Diseases |
| 主題: | |
| オンライン･アクセス: | https://doi.org/10.1186/s13023-024-03108-x |
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