Edgewise Therapeutics Inc at Goldman Sachs Global Healthcare Conference Transcript
All right. Thanks, everyone, for joining us today at the Goldman Sachs Global Healthcare Conference. Really excited to be joined by the team from Edgewise Therapeutics. And let's just dive right in. So I [really wanted] to start the discussion of your lead program 5506 in Becker's muscular dystrophy. I think it's important if you start by walking us through the mechanism of action of 5506 in muscular dystrophies.
Great. Thanks for the invitation. Thank you all for joining today. So let me at the beginning here set the stage for what is really happening in a muscular dystrophy. So muscular dystrophy is a disease where a key structural protein, dystrophin, is missing in patients with what's called Duchenne muscular dystrophy; or that particular protein dystrophin is at very low concentrations and/or mutated in a group of patients called Becker muscular dystrophy.
What's interesting about this space is people have really thought that, well, if I provide
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