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Finnish gelsolin amyloidosis causes significant disease burden but does not affect survival: FIN-GAR phase II study

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 (FIN-GAR)...

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Detalhes bibliográficos
Publicado no:Orphanet J Rare Dis
Main Authors: Schmidt, Eeva-Kaisa, Mustonen, Tuuli, Kiuru-Enari, Sari, Kivelä, Tero T., Atula, Sari
Formato: Artigo
Idioma:Inglês
Publicado em: BioMed Central 2020
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6969418/
https://ncbi.nlm.nih.gov/pubmed/31952544
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-020-1300-5
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