The natural history and burden of illness of metachromatic leukodystrophy: a systematic literature review
Abstract Background Metachromatic leukodystrophy (MLD; OMIM 250100 and 249900) is a rare lysosomal storage disease caused by deficient arylsulfatase A activity, leading to accumulation of sulfatides in the nervous system. This systematic literature review aimed to explore the effect of MLD on the li...
| Principais autores: | , , |
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| Formato: | Artigo |
| Idioma: | English |
| Publicado em: |
BMC
2024-03-01
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| coleção: | European Journal of Medical Research |
| Assuntos: | |
| Acesso em linha: | https://doi.org/10.1186/s40001-024-01771-1 |