Navigating Therapeutic Pathways and Transforming Care in ATTR-CM - Episode 6
Mechanistic Roles of Transthyretin Silencers and Stabilizers in ATTR-CM
The panel reviews the two major therapeutic strategies for treating ATTR-CM: transthyretin (TTR) silencers and stabilizers. Stabilizers work by binding to the TTR protein and preventing it from misfolding into amyloid fibrils, helping maintain the protein’s native structure. Silencers, by contrast, act at the genetic level to reduce the production of TTR protein in the liver through RNA interference or antisense mechanisms. Together, these approaches aim to reduce amyloid formation and slow disease progression. The experts note that patients are often highly informed and eager to understand the differences between these therapies. They emphasize the importance of clear, patient-centered discussions that explain how the treatments work, why both options exist, and how therapy selection may depend on disease stage, phenotype, genetic background, and emerging clinical evidence. As research evolves, clinicians continue to refine how these mechanisms complement one another and how combination or sequential strategies may shape future care.
The panel reviews the two major therapeutic strategies for treating ATTR-CM: transthyretin (TTR) silencers and stabilizers. Stabilizers work by binding to the TTR protein and preventing it from misfolding into amyloid fibrils, helping maintain the protein’s native structure. Silencers, by contrast, act at the genetic level to reduce the production of TTR protein in the liver through RNA interference or antisense mechanisms. Together, these approaches aim to reduce amyloid formation and slow disease progression.
The experts note that patients are often highly informed and eager to understand the differences between these therapies. They emphasize the importance of clear, patient-centered discussions that explain how the treatments work, why both options exist, and how therapy selection may depend on disease stage, phenotype, genetic background, and emerging clinical evidence. As research evolves, clinicians continue to refine how these mechanisms complement one another and how combination or sequential strategies may shape future care.
