Finnish gelsolin amyloidosis causes significant disease burden but does not affect survival: FIN-GAR phase II study
Abstract Background Hereditary gelsolin (AGel) amyloidosis is an autosomal dominantly inherited systemic amyloidosis that manifests with the characteristic triad of progressive ophthalmological, neurological and dermatological signs and symptoms. The National Finnish Gelsolin Amyloidosis Registry (F...
Main Authors: | , , , , |
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Format: | Article |
Language: | English |
Published: |
BMC
2020-01-01
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Series: | Orphanet Journal of Rare Diseases |
Subjects: | |
Online Access: | https://doi.org/10.1186/s13023-020-1300-5 |