Understanding the patient experience of Classic Galactosemia in pediatric and adult patients: increased disease burden, challenges with daily living, and how they evolve over time
Abstract Background Classic Galactosemia (CG) is a rare, autosomal recessive condition. Newborn screening and a timely galactose-restricted diet can resolve acute symptoms and decrease fatalities, but significant chronic, progressive morbidities remain and significantly impact daily life. The object...
| Main Authors: | , , , , , , , |
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| 格式: | 文件 |
| 语言: | English |
| 出版: |
SpringerOpen
2023-09-01
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| 丛编: | Journal of Patient-Reported Outcomes |
| 主题: | |
| 在线阅读: | https://doi.org/10.1186/s41687-023-00635-2 |