Episode 60
Biotech Hangout
00:00
PTC and X4: A Comparison of Drugs and Risk Benefits
Daphne: How clear cut is the data? If it's so clear and so obvious, probably makes the discussion and decision and process easier if you have 50% cure rates. But when you're on the border, it gets a little more murky. And then the safety becomes more murky. When you tie that together to overall risk benefit, you're coming down to a lot of management trusts and then some FDA guessing. The big question will be whether this results translate into broader neutropenia and other like white blood cell issues such as WEN syndrome or WHIM.
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