Prothena Biosciences

Systemic amyloidosis can be hereditary or acquired with autosomal dominant mutations in the transthyretin gene (TTR) being the most common cause of hereditary…

For the first time, we report of a Swedish family of five individuals with a TTR Glu54Leu (p. Glu74Leu) mutation in the transthyretin gene. This mutation has been…

This channel is dedicated to providing resources & information on hATTR amyloidosis.

When Greg first learned about a rare disease affecting his brother, he had no idea that the illness would come to define his own life, or that his search for…

Background: Hereditary transthyretin amyloidosis (hATTR) is associated with significant morbidity and mortality. Early diagnosis and treatment are essential to…

Background: Transthyretin (TTR) amyloidosis is an underdiagnosed disease caused by destabilization of TTR due to pathogenic mutations or aging. Both pathogenic and…

Introduction: Hereditary transthyretin-mediated amyloidosis (ATTRv; v for variant) is an underdiagnosed, progressive, and fatal multisystemic disease with a…

Akcea Therapeutics UK Ltd., has announced that NICE has issued a positive Final Evaluation Document (FED) for Tegsedi(tm) (inotersen) for the treatment of stage 1…

Living with hATTR amyloidosis can cause stress and may make it hard to interact with others. It is important to remember that these are normal feelings following…

Deciding to leave your job following your hATTR amyloidosis diagnosis can be a difficult and personal decision. There is no right or wrong way to go about making…