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Inotersen: new promise for the treatment of hereditary transthyretin amyloidosis
Hereditary transthyretin amyloidosis is a fatal autosomal dominant disorder characterized by deposition of transthyretin amyloid into the peripheral nervous system, heart, kidney, and gastrointestinal tract. Previous treatments using liver transplantation and small molecule stabilizers were not effe...
| Autores principales: | , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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Dove Medical Press
2019
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| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6507904/ https://www.ncbi.nlm.nih.gov/pubmed/31118583 http://dx.doi.org/10.2147/DDDT.S162913 |