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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...

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Publicat a:Drug Des Devel Ther
Autors principals: Mathew, Veena, Wang, Annabel K
Format: Artigo
Idioma:Inglês
Publicat: Dove Medical Press 2019
Matèries:
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC6507904/
https://ncbi.nlm.nih.gov/pubmed/31118583
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.2147/DDDT.S162913
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