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

Hereditary gelsolin (AGel) amyloidosis is an autosomal dominantly inherited systemic amyloidosis that manifests with the characteristic triad of progressive ophthalmological, neurological and dermatological si...

Source: Orphanet Journal of Rare Diseases - January 17, 2020 Category: Internal Medicine Authors: Eeva-Kaisa Schmidt, Tuuli Mustonen, Sari Kiuru-Enari, Tero T. Kivel ä and Sari Atula Tags: Research Source Type: research