Familial amyloidosis of the Finnish type: clinical and neurophysiological features of two index cases.
Inês Antunes CunhaAna BrásFátima SilvaAnabela MatosPublished in: BMJ case reports (2022)
Familial amyloidosis of the Finnish type (FAF) is a rare multisystemic disorder caused by mutations in the gelsolin gene. The clinical presentation is typically characterised by a triad of ophthalmic, neurological and dermatological findings. FAF has been reported in several countries, primarily in Finland and recently in Portugal. We report the first genetically confirmed cases of FAF from two unrelated families in our neuromuscular outpatient clinic. Gelsolin gene sequencing revealed the heterozygous gelsolin mutation (c.640G>A). The clinical features and the neurophysiological studies of two index patients and their relatives are presented. Obtaining an early diagnosis can be challenging, but FAF should be considered in the differential diagnosis of progressive bilateral facial neuropathy, even if there is no known Finnish ancestor.
Keyphrases
- early onset
- end stage renal disease
- ejection fraction
- chronic kidney disease
- newly diagnosed
- copy number
- single cell
- genome wide
- multiple sclerosis
- peritoneal dialysis
- primary care
- multiple myeloma
- prognostic factors
- genome wide identification
- patient reported outcomes
- gene expression
- dna methylation
- brain injury
- transcription factor