We spoke to Professor Morie Getz at ASH 2018 about the current unmet needs in treatment of hereditary TTR amyloidosis, antisense technology, inotersen and the NEURO-TTR study.
Questions:
1. What is hereditary TTR amyloidosis and what are the unmet needs in its treatment? (0:10)
2. Could you tell us a little about antisense technology and the mechanism of action of inotersen? (1:13)
3. What have been the major efficacy and safety findings of the NEURO-TTR study to date? (2:10)
4. What were the conclusions of the recent long-term update from the open-label extension of NEURO-TTR? (2:58)
5. What are the next steps in the clinical development of inotersen? (3:41)
Speaker’s disclosures: Morie A Gertz has received honoraria from Akcea and Ionis.
Filmed at the 60th American Society of Hematology (ASH) 2018 Annual Meeting, San Diego, CA, US, December 1–4, 2018.
