Recent Advances in the Treatment of Hereditary ATTR Amyloidosis (hATTR) - Episode 13

Benefits of TTR Gene Silencers

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Discussion on the role of TTR protein stabilizers and gene silencers for treating hereditary ATTR amyloidosis.

John L. Berk, MD: Dr Polydefkis, Dr Witteles suggested that the earlier the better with most effective therapy, alluding to TTR gene silencers. Let’s say I have fairly advanced, previously undiagnosed TTR amyloid neuropathy. I’m seeking your advice; am I out of luck in terms of getting benefits from the TTR gene silencers?

Michael J. Polydefkis, MD: No. It’s been shown that irrespective of neuropathy severity, you’ll still experience a benefit from silencer therapy. This means that your disease will be likely halted. You may not improve dramatically, you may not go from a cane to running marathons, but you won’t regress to 2 canes or to a wheelchair.

John L. Berk, MD: Thank you for watching this NeurologyLive® Peer Exchange. If you enjoyed the content, please subscribe to our e-newsletters to receive upcoming Peer Exchanges and other great content right in your inbox.

Transcript Edited for Clarity