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- URN zum Zitieren dieses Dokuments:
- urn:nbn:de:bvb:355-epub-505481
- DOI zum Zitieren dieses Dokuments:
- 10.5283/epub.50548
Zusammenfassung
Hereditary transthyretin amyloidosis is caused by pathogenic variants (ATTR(v)) in the TTR gene. Alongside cardiac dysfunction, the disease typically manifests with a severely progressive sensorimotor and autonomic polyneuropathy. Three different drugs, tafamidis, patisiran, and inotersen, are approved in several countries, including the European Union and the United States of America. By ...
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