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Ethan Perlstein 1-to-N
62.3K posts
user avatar
Ethan Perlstein 1-to-N
@eperlste
ceo @1000cures, ceo @PerlaraPBC (w16 @ycombinator), founder @epalrestat, founder @endrarediseases, founder @smer28rapa
Bicoastal
1000cures.com
Joined January 2011
1,833
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19.1K
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  • Pinned
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    Ethan Perlstein 1-to-N
    @eperlste
    Apr 17
    I've met so many entrepreneurial rare disease parents over the years that I’ve lost count. They have moved or are moving medical mountains to save their kids and others like them. But rare parent founders get no love from institutional players. Those days are done. Science,
    187K
  • user avatar
    Ethan Perlstein 1-to-N
    @eperlste
    Jul 16, 2023
    Replying to @RyanFMandelbaum
    If libraries were smart they’d all have popup cafes and support a popup ecosystem! Why does it have to be one (legacy choice) or the other?
    755K
  • user avatar
    Ethan Perlstein 1-to-N
    @eperlste
    Feb 28, 2019
    It is with a broken heart but defiant spirit that I announce the wind down of @PerlaraPBC. After 5 unforgettable years this ambitious, hopeful, necessary experiment is over. We lived as the first biotech public benefit company and we die as the first biotech PBC.
  • user avatar
    Ethan Perlstein 1-to-N
    @eperlste
    Sep 17, 2019
    So excited to announce my next patient-inspired adventure as Chief Scientific Officer @ReeveFoundation!! prnewswire.com/news-releases/… It’s time to retire the wheelchair. I can’t wait to help translate basic science into new treatments and ultimately a cure for spinal cord injury!
    Christopher & Dana Reeve Foundation appoints Ethan Perlstein, Ph.D., as Chief Scientific Officer
    From prnewswire.com
  • user avatar
    Ethan Perlstein 1-to-N
    @eperlste
    Jul 27, 2020
    It’s only n-of-1 (as of now) but I still can’t totally wrap my head around the concept that we discovered a drug that’s working in an actual human being! And she’s the first child with her rare disease to ever take this drug. I just want to do it again, and again, and again..
  • user avatar
    Ethan Perlstein 1-to-N
    @eperlste
    Aug 29, 2019
    We welcomed baby Luca to the world at 10:55am PT this morning!!! 6 lbs 7 ounces Scheduled C section went perfectly. Thanks Kaiser Walnut Creek docs and nurses! Mom is recovering. And Kai is excited to finally be a big brother.
  • user avatar
    Ethan Perlstein 1-to-N
    @eperlste
    Jan 19, 2022
    Are there any scientists left in academia after today’s announcement?
  • user avatar
    Ethan Perlstein 1-to-N
    @eperlste
    Jan 5, 2022
    How it started (2014): How it's going (2022):
  • user avatar
    Ethan Perlstein 1-to-N
    @eperlste
    Apr 15, 2023
    Someday grad students will realize that being a postdoc sucks unless you’re academia or bust. The market will reward PhDs with great salaries and great science. There’s no reason to do a postdoc unless you want to be a professor.
    user avatar
    Leslie Vosshall PhD
    @leslievosshall
    Apr 13, 2023
    🧵📢 We need to retain talented postdocs in academic science. I'm happy to say that @HHMINEWS has taken a big step forward this week. $70,000 That's the new starting minimum salary for all postdocs employed in HHMI labs hhmi.org/sites/default/… (1/4)
    469K
  • user avatar
    Ethan Perlstein 1-to-N
    @eperlste
    Dec 29, 2021
    STUDY MAY PROCEED!!!
  • user avatar
    Ethan Perlstein 1-to-N
    @eperlste
    Feb 28, 2019
    Replying to @eperlste
    As one of our dying acts, I am open-sourcing all data, discoveries from the #NGLY1 PerlQuest which was denied a commercial home, starting with this preprint describing 3 therapeutic approaches: NRF2, catecholamine and anti-inflammatory pathway activation
    biorxiv.org
    Drug screens of NGLY1 Deficiency worm and fly models reveal catecholamine, NRF2 and anti-inflamma...
    N -glycanase 1/ NGLY1 Deficiency is an ultra-rare and complex monogenic glycosylation disorder that affects fewer than 40 patients globally. NGLY1 Deficiency has been studied in model organisms such...
  • user avatar
    Ethan Perlstein 1-to-N
    @eperlste
    Aug 30, 2018
    Hey @bloomberg what kind of bullshit sexism is this? You list me as Dr but my PhD colleagues @NinaDiPrimio and @IyerSangeetha are listed as Ms? (h/t @jessplao)
  • user avatar
    Ethan Perlstein 1-to-N
    @eperlste
    Dec 31, 2019
    A year ago we were fundraising for @PerlaraPBC’s Series B. I failed my team and our partners. I couldn’t tell a story that excited VCs. Now that I’m firmly settled and over the moon in my new role — and I’ve had a year to reflect — here’s the deck, with lessons learned:
  • user avatar
    Ethan Perlstein 1-to-N
    @eperlste
    Dec 30, 2021
    Yesterday FDA said the three magic words: Study May Proceed Here’s how we went from a hit in yeast and worm drug repurposing screens to a single-patient IND study to the start of a pivotal Phase 3 in less three years..

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