Are we creating a new phenotype? Physiological barriers and ethical considerations in the treatment of hereditary transthyretin-amyloidosis

Hereditary transthyretin (TTR) amyloidosis (ATTRv) is an autosomal dominant, systemic disease transmitted by amyloidogenic mutations in the TTR gene. To prevent the otherwise fatal disease course, TTR stabilizers and mRNA silencing antisense drugs are currently approved treatment options. With 90% o...

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Detalles Bibliográficos
Autores principales: Dohrn, Maike F., Medina, Jessica, Olaciregui Dague, Karmele R., Hund, Ernst
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2021
Materias:
Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8559355/
https://www.ncbi.nlm.nih.gov/pubmed/34719408
http://dx.doi.org/10.1186/s42466-021-00155-8

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8559355/
https://www.ncbi.nlm.nih.gov/pubmed/34719408
http://dx.doi.org/10.1186/s42466-021-00155-8

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