The headline is broadly defensible, but the qualifications matter. Effect sizes vary by population, the strongest claims rest on shorter trials, and credible voices push back on how it's typically framed.
the cost with Gene therapies is prohibitive right now right it's not as if we could if any single gene therapy were $3 million which is not uncommon for current Gene therapies we can't afford to spend $3 million per person uh with the number of people who are at R in the that SMA single gene therapy is about a million treatment so I'll say the range of genetic therapies right now runs between about1 and $3 million
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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.
Because today, a gene therapy for rare diseases can be a half a million dollars to a couple million dollars. But it turns out that if you look at what Moderna and Pfizer did, it effectively is a type of gene therapy. It's microsomal lipid capsules, with mRNA in them. And what we've seen is that if you do it at a high enough scale, the gene therapy gets down to 20 bucks.