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Eiger BioPharmaceuticals Inc, Applied Therapeutics, Ultragenyx Pharmaceutical, Dicerna Pharmaceuticals, and Imara Inc at Citi BioPharma Conference (Virtual) - Panel Transcript

Sep 09, 2020 / 08:15PM GMT
Yigal Dov Nochomovitz
Citigroup Inc., Research Division - Director

I hope everyone can hear me. Welcome to the session on challenges and opportunity of rare disease drug development. I'm Yigal Nochomovitz, I'm the biotech analyst at Citi. As always, if you have questions during the session, you're welcome to email me at [email protected] or showcase feature on the video to assist in answering questions that way.

So it's my great pleasure to have with me this afternoon 5 English CEOs of rare disease companies. From Applied Therapeutics, Shoshana Shendelman, CEO; from Dicerna, Doug Fambrough, CEO as well; from Eiger, David Cory, President and CEO; from IMARA, Rahul Ballal, CEO; and from Ultragenyx, Emil Kakkis, President and CEO.

So thank you all so much for joining this afternoon.

Questions & Answers

Yigal Dov Nochomovitz
Citigroup Inc., Research Division - Director

Maybe as a -- just as a broad high-level question. I'd love to get your thoughts on design considerations for rare disease trials. What should be avoided when

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