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PMM2-CDG pioneer Maggie has been on epalrestat for almost two years as part of an ongoing single-patient IND study.
Her first dose was January 2020. See the results with your own eyes 👇
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Ethan Perlstein 1-to-N
62.1K posts
Ethan Perlstein 1-to-N
@eperlste
ceo @PerlaraPBC (@ycombinator W16), founder @epalrestat, ceo @endrarediseases $CURES, evo pharmacologist, mTOR worshipper $RAPTOR, $SASHA, cofounder of K&L
Bicoastal Katılım Ocak 2011
1.8K Takip Edilen19K Takipçiler
AI + drug repurposing = derisked drug discovery
Jay Bhattacharya, MD, PhD@NIHDirector_Jay
NIH is proudly prioritizing drug repurposing as a way to accelerate research while using resources efficiently. Watch for some outstanding examples of how we’ve applied gold standard science to find new ways existing therapies can benefit more patients now:
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💊 STUDY MAY PROCEED ✅
First N-of-1 IND has been greenlit, now waiting for word on a second N-of-1 IND.
This is for a repositioned investigational drug for two patients with a rare inherited metabolic disease.
First-patient, first-dose coming soon..
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@SimonDBarnett @GaryMarcus Would you be so bold as to call out the clearest examples of over-promise and under-delivery?
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I can feel @GaryMarcus’s frustration. AI’s utility—specifically in drug development—was over-promised and under-delivered upon.
But I still can’t help but get annoyed at the concept of an AI drug. They don’t exist and I look forward to when we stop using this phrase.
AI is a utility. It’s a raw resource. It’s intelligence in a box (er, data center).
Electricity is the same way. It gets pushed into devices that transform that utility into another unit of work.
AI gets pushed into different nodes of the drug discovery paradigm and also gets transformed into units of work.
That’s why the phrase is based entirely on a false premise.
If we were talking about building bridges. We wouldn’t have this issue. Is electricity used to build bridges? Sure! There are all sorts of electrically-powered tools used to build bridges. Small ones. Big ones.
But is it an electrically-built bridge? Do 51% of the rivets need to get installed with electricity and not gasoline as the raw fuel?
What about drug development? Do 51% of the atoms require data-driven models versus human heuristics and/or physics?
I don’t know!
The phrase AI drug is an allergic reaction to the irrational over-hyping of an idea and the ensuing misallocation of investor capital. It is rarely used in a scientific or technical sense.
Nowadays, it’s mostly used by some who tacitly root on the missteps or failures of so-called AI drugs — as a bit of ‘I told you so’.
And I get it! It’s well placed and somewhat deserved, but doesn’t move the needle on scientific discourse.
Gary Marcus@GaryMarcus
F Cancer Why has AI had so little impact on Cancer? New essay, link below.
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To be clear, by robust, I mean the public conversation on Twitter is broad & so is deliberation/engagement from industry. As the person who is often on the itty bitty conference call, WG, or niche panel on this subject, I'm just like.... really, the dog is what got ppl talking?
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This dog has spurred public convos about evidence generation, regulation, and economics of individualized therapies 10x more robust than Baby KJ or Mila. Is it just that the "sidequest" nature of the story is making folks internalize that tech is eons ahead of the rest?
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@ethanjweiss Yeah, same glitch. It was back a few minutes later
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Anyone just lose their internet? Was on a zoom with 5 people and 3 of them just lost it at same time
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@eperlste Wondering, how do you connect with rare disease parents in the first place?
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Ethan Perlstein 1-to-N retweetledi
If you’re blown away by the lengths a person will go to save their dog, let me introduce you to every rare disease parent I’ve ever met.
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People quitting AI because of its use of water should get the career version of the Darwin Award. We should thank them for removing their bad judgement out of the workforce.
Andy Masley@AndyMasley
I've been summoned (about this article specifically). Here's a quick thread on why this is making the same bizarre moves as all the others. As Rowland said above, I don't know why so many people seem so intent to overstate weak evidence. theguardian.com/australia-news…
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People (myself included) have been underestimating the “consumer scientist” demand. The two big blockers today to at-home science are:
1. Esoteric knowledge - AI models knock it down.
2. Lab access - cloud autonomous labs knock it down.
Let’s get going.
vittorio@IterIntellectus
this is actually insane > be tech guy in australia > adopt cancer riddled rescue dog, months to live > not_going_to_give_you_up.mp4 > pay $3,000 to sequence her tumor DNA > feed it to ChatGPT and AlphaFold > zero background in biology > identify mutated proteins, match them to drug targets > design a custom mRNA cancer vaccine from scratch > genomics professor is “gobsmacked” that some puppy lover did this on his own > need ethics approval to administer it > red tape takes longer than designing the vaccine > 3 months, finally approved > drive 10 hours to get rosie her first injection > tumor halves > coat gets glossy again > dog is alive and happy > professor: “if we can do this for a dog, why aren’t we rolling this out to humans?” one man with a chatbot, and $3,000 just outperformed the entire pharmaceutical discovery pipeline. we are going to cure so many diseases. I dont think people realize how good things are going to get
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Swallowell now has the attention of all of his opponents, and the smearing has began.
If he somehow makes it through it all, CA is def F'd in the you know what.
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Austin might be the least sexy city I’ve been to in America. Right up there with Charlotte, NC
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@ajhodls because vc is a commodity now: a few top-shelf brands floating in a sea of undifferentiated competing options
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Ethan Perlstein 1-to-N retweetledi
Aging may feel gradual… but what if it’s not?
In our paper out today, we tracked fish continuously from puberty until death.
This gave us a unique view of how aging unfolds across the adult lifespan.
🧵
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@linderps balbs does more lifting than any other bar in a city as big as sf. very fitting for a town built on winner-take-all network effects
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delete dating apps.
join the run club.
go to dinner parties.
join a silly YC founder mixer.
heck, go to balboa if you must.
just start meeting people in real life again
Hank@HankFrank
Deleted the dating apps. Joined a run club. Met her on a random Wednesday night run. Moved in together 10 months later. She’s my best friend.
San Francisco, CA 🇺🇸 English
in sf rn two companies are locked in hand to hand mortal combat
no, not openai and anthropic
hinge vs raya
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since there’s not enough violence in the world at the moment already
Anish Koka, MD@anish_koka
Better things to do than spend Saturday morning writing this, but important since there aren't enough actual journalists who can do their job. (link in reply)
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