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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Shranjeno v:
Bibliografske podrobnosti
izdano v:Orphanet J Rare Dis
Main Authors: Schmidt, Eeva-Kaisa, Mustonen, Tuuli, Kiuru-Enari, Sari, Kivelä, Tero T., Atula, Sari
Format: Artigo
Jezik:Inglês
Izdano: BioMed Central 2020
Teme:
Research
Online dostop: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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