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Therapeutic landscape for Batten disease: current treatments and future prospects
Batten disease (also known as neuronal ceroid lipofuscinoses) constitutes a family of devastating lysosomal storage disorders that collectively represent the most common inherited paediatric neurodegenerative disorders worldwide. Batten disease can result from mutations in 1 of 13 genes. These mutat...
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| Pubblicato in: | Nat Rev Neurol |
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| Autori principali: | , , , , , |
| Natura: | Artigo |
| Lingua: | Inglês |
| Pubblicazione: |
2019
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| Soggetti: | |
| Accesso online: | https://ncbi.nlm.nih.gov/pmc/articles/PMC6681450/ https://ncbi.nlm.nih.gov/pubmed/30783219 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41582-019-0138-8 |
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