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ISA 2024 | Knowledge gaps in our understanding of the pathophysiology of ATTR amyloidosis
Lorena Saelices Gomez, PhD, UT Southwestern Medical Center, Dallas, TX, highlights the main gaps in our understanding of the pathophysiology of transthyretin (ATTR) amyloidosis. First, the differential diagnosis of ATTR amyloidosis is quite challenging, as patients present with variable clinical manifestations. Secondly, how, where, and when these fibrils are formed is still unknown. Thirdly, it is still unclear whether amyloidosis is a disease of aggregation or impaired clearance. Despite these knowledge gaps, several studies are being conducted to better understand this disease and diagnose it at an earlier stage. This interview took place at the XIX International Symposium on Amyloidosis (ISA) in Rochester, MN.
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Disclosures
Honoraria for scientific consulting for Intellia and Attralus
Member of the advisory board of Alexion
