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Combination Therapies for Lysosomal Storage Diseases: A Complex Answer to a Simple Problem
Lysosomal storage diseases (LSDs) are a group of 40–50 rare monogenic disorders that result in disrupted lysosomal function and subsequent lysosomal pathology. Depending on the protein or enzyme deficiency associated with each disease, LSDs affect an array of organ systems and elicit a complex set o...
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| Publicado no: | Pediatr Endocrinol Rev |
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| Autor principal: | |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
2016
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC5374980/ https://ncbi.nlm.nih.gov/pubmed/27491211 |
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