The parent and family impact of CLN3 disease: an observational survey-based study
Abstract Background CLN3 disease (also known as CLN3 Batten disease or Juvenile Neuronal Ceroid Lipofuscinosis) is a rare pediatric neurodegenerative disorder caused by biallelic mutations in CLN3. While extensive efforts have been undertaken to understand CLN3 disease etiology, pathology, and clini...
| Main Authors: | , , , , , |
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| 格式: | 文件 |
| 语言: | English |
| 出版: |
BMC
2024-03-01
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| 丛编: | Orphanet Journal of Rare Diseases |
| 主题: | |
| 在线阅读: | https://doi.org/10.1186/s13023-024-03119-8 |