Transthyretin

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Okay, take 2.

Hello, I am a student shadowing in Cardiology. The physician I am shadowing gave me an assignment, to investigate current therapeutic strategies for the management of Transthyretin Cardiomyopathy. Now, I have scoured the literature up and down, and it appears that this diagnosis is essentially a death sentence. All sources indicate that management should be targeted at 1. Treatment of Symptoms and 2. Reduction of precursor proteins to amyloid, which in the case of TTR cardiomyopathy, would be liver transplant as a form of surgical gene therapy. I am here, to learn, if anyone knows of any studies, clinical trials, or established protocols that have shown promise or have achieved goal #2, as listed above?

I appreciate the input, thank you in advance.
 
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