The evidence is convergent. Multiple independent sources reach the same conclusion, the underlying mechanism is well-characterized, and even the field's most cautious voices treat it as worth doing.
even over the course of this study the sequencing costs have come down if you can imagine it and we just started it like I said September 2022 less than a year ago but already the sequencing costs have come down I expect they'll continue to come down
Every Sunday: the week’s new conflicts and verdict changes — and nothing else.
Native comments, Twitter mentions, and Reddit threads about this claim — surfaced together so the conversation isn't fragmented across platforms.
Bookmarking — the dossier-vs-overview split is the right call. Most of the time I want overview; sometimes I want receipts.
Would love a "what would change this verdict" RSS feed. Sign me up if it exists.
so as we started out doing this round number $1,000 per baby so thinking about generating the data interpreting the data getting it back to folks cost of about $1,000 per baby
so at the outset of this discussion you mentioned that SMA actually has a successful gene therapy I was not aware of that yes um how many of these um uh single Gene highly penetrant conditions that that you know children are born with be it inborn eras of metabolism or neurod degener diseases Etc how many of them have FDA approved Gene therapies already
the goal is to be able to do this and get it down by the order of magnitude can we get it down to $100 per baby as an example