Gene Therapy for hemophilia must wait, for now.

To the surprise of many, on August 19th the US Food and Drug Administration denied the first application for a one-time gene therapy for hemophilia A. Patrick and Amy process this major news then share soundbites from their conversations with doctors Valentino, Quon, Young, and Wang during #NHF2020's Science Fair about gene therapy, emicizumab, and novel therapies.

Show Notes:

Presenting Sponsor: Takeda

Response to FDA’s Actions: HFA Statement

The Science Fair is made possible by Spark Therapeutics