Healthcare utilization and disease burden in children with metachromatic leukodystrophy in Germany
Abstract Background Metachromatic leukodystrophy (MLD) is a life-limiting neurodegenerative disease due to pathogenic variants in the ARSA gene. Patients experience severe neurological symptoms, developmental regression, and early death. Aim of the study was to analyze disease burden and healthcare...
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| Main Authors: | , , , , , , |
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| Format: | Article |
| Language: | English |
| Published: |
BMC
2025-05-01
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| Series: | Orphanet Journal of Rare Diseases |
| Subjects: | |
| Online Access: | https://doi.org/10.1186/s13023-025-03637-z |
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