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Novel RNA-targeted therapies for hereditary ATTR amyloidosis and their impact on the autonomic nervous system
PURPOSE: Transthyretin-mediated hereditary amyloidosis (hATTR amyloidosis) is a multisystemic disease with heterogeneous clinical presentation. Hallmarks of the disease are sensory-motor and autonomic neuropathy and cardiomyopathy. Two disease-modifying drugs, inotersen (an antisense oligonucleotide...
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| Udgivet i: | Clin Auton Res |
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| Hovedforfatter: | |
| Format: | Artigo |
| Sprog: | Inglês |
| Udgivet: |
Springer Berlin Heidelberg
2019
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| Fag: | |
| Online adgang: | https://ncbi.nlm.nih.gov/pmc/articles/PMC6763402/ https://ncbi.nlm.nih.gov/pubmed/31399774 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/s10286-019-00626-8 |
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