Friday, December 14, 2018 10:40:49 AM
Maybe Buerger's Disease would have been the ticket. Taking the 'ultra-orphan' route in rare diseases has commanded drug prices of over $1M per patient/annually. Eleprase, etc. in the enzyme replacement market.
In the case of ALXN and PNH, TX cost is well north of 500K/PT/Year...
Coulda, Should, Would, but the 3D bet is yet to be covered.
I do fear the loss of any head-start in this space.
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