New insights into the clinical evaluation of hereditary transthyretin amyloidosis patients: a single center's experience.

ABSTRACT: Over the last decade, new medical treatment modalities have emerged based on increased insights into amyloid formation. With the increased possibilities for treatment of amyloidosis caused by transthyretin (TTR) amyloid deposits comes the need for diagnostic procedures for early diagnosis and better tools to follow disease progression. This is of particular importance in clinical trials evaluating the efficacy of new treatments. Until recently, the treatment of TTR amyloidosis (ATTR) was based solely on liver transplantation, a procedure that has halted disease progression in many patients. Liver transplantation has been especially effective in patients under the age of 50 years carrying the TTR V30M mutation, whereas the outcome of the procedure has been variable for others, particularly

SUBMITTER: Suhr OB 

PROVIDER: S-EPMC6065582 | biostudies-literature | 2012

REPOSITORIES: biostudies-literature