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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Enregistré dans:
Détails bibliographiques
Publié dans:Orphanet J Rare Dis
Auteurs principaux: Schmidt, Eeva-Kaisa, Mustonen, Tuuli, Kiuru-Enari, Sari, Kivelä, Tero T., Atula, Sari
Format: Artigo
Langue:Inglês
Publié: BioMed Central 2020
Sujets:
Research
Accès en ligne: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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