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Patisiran in hATTR Amyloidosis: Six-Month Latency Period before Efficacy
Hereditary amyloidosis associated with mutations in the transthyretin gene (hATTR) is a progressive devastating disease, with a fatal outcome occurring within 10years after onset. In recent years, TTR gene silencing therapy appeared as a promising therapeutic strategy, showing evidence that disease...
Αποθηκεύτηκε σε:
| Τόπος έκδοσης: | Brain Sci |
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| Κύριοι συγγραφείς: | , , , , , , , , , , , |
| Μορφή: | Artigo |
| Γλώσσα: | Inglês |
| Έκδοση: |
MDPI
2021
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| Θέματα: | |
| Διαθέσιμο Online: | https://ncbi.nlm.nih.gov/pmc/articles/PMC8073153/ https://ncbi.nlm.nih.gov/pubmed/33921571 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/brainsci11040515 |
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