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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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Detalles Bibliográficos
Autores principales: Schmidt, Eeva-Kaisa, Mustonen, Tuuli, Kiuru-Enari, Sari, Kivelä, Tero T., Atula, Sari
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2020
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6969418/
https://www.ncbi.nlm.nih.gov/pubmed/31952544
http://dx.doi.org/10.1186/s13023-020-1300-5