Biophysical characterization and modulation of Transthyretin Ala97Ser.

Yo-Tsen LiuYueh-Jung YenFrans RicardoYu ChangPei-Hao WuShing-Jong HuangKon-Ping LinTsyr-Yan Yu

Published in: Annals of clinical and translational neurology (2019)

This study confirmed the structural modulation effect of tafamidis on A97S-TTR and implied the potential therapeutic benefit of tafamidis for A97S TTR-FAP. This approach can be applied to investigate the modulation effect of tafamidis on other rare TTR variants and help to make individualized choices of available treatments for FAP patients.

Keyphrases

end stage renal disease
ejection fraction
newly diagnosed
chronic kidney disease
peritoneal dialysis
gene expression
patient reported outcomes
human serum albumin