A phase II, open-label, extension study of long-term patisiran treatment in patients with hereditary transthyretin-mediated (hATTR) amyloidosis

BACKGROUND: Patisiran, an RNA interference therapeutic, has demonstrated robust reduction of wild-type and mutant transthyretin protein and was able to improve polyneuropathy and quality of life following 18 months of treatment in patients with hereditary transthyretin-mediated (hATTR) amyloidosis....

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Bibliografiset tiedot
Julkaisussa:Orphanet J Rare Dis
Päätekijät: Coelho, Teresa, Adams, David, Conceição, Isabel, Waddington-Cruz, Márcia, Schmidt, Hartmut H., Buades, Juan, Campistol, Josep, Berk, John L., Polydefkis, Michael, Wang, Jing Jing, Chen, Jihong, Sweetser, Marianne T., Gollob, Jared, Suhr, Ole B.
Aineistotyyppi: Artigo
Kieli:Inglês
Julkaistu: BioMed Central 2020
Aiheet:
Research
Linkit:https://ncbi.nlm.nih.gov/pmc/articles/PMC7341568/
https://ncbi.nlm.nih.gov/pubmed/32641071
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-020-01399-4
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