Amyloidosis News has launched a 9-part series of video clips with top experts in the field of amyloidosis research using its “Interview with the Innovators” platform. The series focuses on the role of a protein called transthyretin, or TTR in normal physiology, the pathophysiology of TTR amyloidosis (ATTR), the potential clinical implications of novel disease-specific therapies, and the role of serum TTR as a biomarker.

Advances in our knowledge of how complex diseases such as amyloidosis develop and progress over time are fundamental in the development of better therapies. Over the past few years, key information has emerged on the crucial role that TTR plays in this disease. The studies that led to our enhanced understanding of ATTR as a protein misfolding disorder were conducted across many disciplines, such as genetics and genomics (ie, phenotype to gene), reverse genetics (ie, gene to phenotype), biochemistry, molecular biology, and translational science (ie, studies on the formation of abnormal deposition of amyloid in the organs and tissues of affected patients). All of the above information contributed to rational drug design approaches that directly target the purported mechanism of the disease, or MOD in amyloidosis, leading to clinical studies with novel disease-specific medicines.