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Pharmacokinetics of Patisiran, the First Approved RNA Interference Therapy in Patients With Hereditary Transthyretin‐Mediated Amyloidosis

Hereditary transthyretin‐mediated (hATTR) amyloidosis is a rare, inherited, progressively debilitating, and often fatal disease caused by deposition of mutated transthyretin (TTR) protein. Patisiran is an RNA interference therapeutic comprising a novel small interfering ribonucleic acid (ALN‐18328)...

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Vydáno v:J Clin Pharmacol
Hlavní autoři: Zhang, Xiaoping, Goel, Varun, Robbie, Gabriel J.
Médium: Artigo
Jazyk:Inglês
Vydáno: John Wiley and Sons Inc. 2019
Témata:
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC7187331/
https://ncbi.nlm.nih.gov/pubmed/31777097
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/jcph.1553
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