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Msg  167991 of 168914  at  9/29/2022 8:15:32 PM  by

rynotheknife

The following message was updated on 9/29/2022 8:25:01 PM.

 In response to msg 167988 by  ZFP
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Re: Fabry recap

that "fireside chat" was a Q&A between the analyst and McClung... and while the analyst tried to push accelerated approval at every opportunity, McClung definitely downplayed it

yes, and substrate reduction was gonna be the surrogate endpoint for AVRO's planned application for an accelerated approval application but that pathway closed after Fabrazyme was granted full approval... and BTW, AVRO showed 87% and 100% substrate reduction for its 2 patients who had kidney biopsies but (as bct1192 so diligently reminds us) that trial crashed and burned... but that could never happen to SGMO because SM says "Fabry will be a medicine" :-)

and McClung said the ESCGT data has a July 20 (or 21) cutoff date... so the 5e13 cohort patients (who were both ERT naive) will have ~3 months follow-up... that's hardly "early" as compared to patient 6 who had only 2 weeks of follow-up at SSIEM


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Msg # Subject Author Recs Date Posted
167993 Re: Fabry recap ZFP 2 9/29/2022 10:23:32 PM




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