A retrospective survey of patients with hereditary transthyretin-mediated (hATTR) amyloidosis treated with patisiran in real-world clinical practice in Belgium.
Hereditary transthyretin-mediated (hATTR) amyloidosis, a genetic disease caused by mutations in the transthyretin gene, leads to progressive sensory and autonomic neuropathy and/or cardiomyopathy and is associated with renal and ophthalmologic manifestations and a poor prognosis. ; SCOPUS: ar.j ; info:eu-repo/semantics/published
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Dokumenttyp: | Artikel |
Erscheinungsdatum: | 2023 |
Schlagwörter: | Sciences bio-médicales et agricoles / Humans / Amyloid Neuropathies / Familial -- complications -- drug therapy -- genetics / Retrospective Studies / Belgium / Prealbumin -- genetics / Polyneuropathies -- etiology / Patisiran / Polyneuropathy / Real-world evidence / hATTR |
Sprache: | Englisch |
Permalink: | https://search.fid-benelux.de/Record/base-28538921 |
Datenquelle: | BASE; Originalkatalog |
Powered By: | BASE |
Link(s) : | http://hdl.handle.net/2013/ULB-DIPOT:oai:dipot.ulb.ac.be:2013/365356 |