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 (FIN-GAR) was founded in 2013 to collect clinical data on patients with AGel amyloidosis, including altogether approximately one third of the Finnish patients. We aim to deepen knowledge on the disease burden and life span of the patients using data from the updated FIN-GAR registry. We sent an updated questionnaire concerning the symptoms and signs, symptomatic treatments and subjective perception on disease progression to 240 members of the Finnish Amyloidosis Association (SAMY). We analyzed the

SUBMITTER: Schmidt EK 

PROVIDER: S-EPMC6969418 | biostudies-literature | 2020 Jan

REPOSITORIES: biostudies-literature