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

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...

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Podrobná bibliografie
Hlavní autoři: Eeva-Kaisa Schmidt, Tuuli Mustonen, Sari Kiuru-Enari, Tero T. Kivelä, Sari Atula
Médium: Článek
Jazyk:English
Vydáno: BMC 2020-01-01
Edice:Orphanet Journal of Rare Diseases
Témata:
Gelsolin
Amyloidosis
AGel
Hereditary amyloidosis
Meretoja syndrome
Natural history
On-line přístup:https://doi.org/10.1186/s13023-020-1300-5

Internet

https://doi.org/10.1186/s13023-020-1300-5

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