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A charitable access program for patients with lysosomal storage disorders in underserved communities worldwide
BACKGROUND: Lysosomal storage disorders (LSDs) are rare genetic disorders, with heterogeneous clinical manifestations and severity. Treatment options, such as enzyme replacement therapy (ERT), substrate replacement therapy, and pharmacological chaperone therapy, are available for several LSDs, inclu...
| Autores principales: | , , , , , , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
BioMed Central
2021
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| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7788852/ https://www.ncbi.nlm.nih.gov/pubmed/33407729 http://dx.doi.org/10.1186/s13023-020-01645-9 |