Van Duesterberg PhD

Patient Scientist Discovering a Cure For My Blindness and Helping Others While I Am At It. Postdoc @UCSF. Follow my journey at

[Ethan Perlstein 1-to-N @eperlste]

Early promising results from our latest drug repurposing n-of-1 study after less than a week of treatment!

The top hit is a generic drug we discovered in a fly screen with @ClementYChow. We've translated directly from flies to kiddos twice so far.

We're just getting started..

[Ethan Perlstein 1-to-N @eperlste]

Want to see what a direct-to-patient biotech pipeline looks like?

22 programs funded by pioneer families, 12 in various stage of 1-to-N clinical studies, and half of hits are clinically actionable nutraceuticals.

This is how you start to do drug repurposing at scale.

[Van Duesterberg PhD @vancanwin]

I got be part of a cure odyssey for @sn_telesca. I can’t imagine the historical implications for channelopathies and how we found several dozen possible treatments through drug repurposed screening with @MetrionBio and @wldolan. @PerlaraPBC. Read more https://t.co/gJRbk1b4BV

[Ethan Perlstein 1-to-N @eperlste]

Whoa @PerlaraPBC is about to surpass $1M in ARR, bootstrapping from $0 in 2021.

Credit goes to our amazing distributed team: Mimi Tran, @vancanwin, @wolfeofscience, @JDon_chembio and James Joanani.

Let’s go!

[Ethan Perlstein 1-to-N @eperlste]

A new 1-to-N drug repurposing study popped into existence over the weekend based on results from yeast avatar screens.

Here’s the note we just got from the dad:

“Hi everyone! On Saturday morning we started XXXXX on XXXXX in daily doses of XXX micrograms. Since then, they have gone from having 3 or 4 dystonic seizures a day to having only one of short duration last night.

They had been in this state for months and unless it was a coincidence, it seems that the XXXXX has been the cause of the improvement!”

[Van Duesterberg PhD @vancanwin]

@eperlste Again amazing.

[Van Duesterberg PhD @vancanwin]

@ALiS0NLAURA I still have hip pain from my pregnancy 3 years ago and no one was able to help me until I figured out what exactly helped. The lack of womens anatomy and biology in medicine is baffling.

[Van Duesterberg PhD @vancanwin]

@ALiS0NLAURA Oh on the contrary, Mother Earth will survive. She’s just sick of tired of cleaning up her kids mess and just letting them deal with it. In the end, human kind will cease to exist and she will continue to harbor different life forms.

[Van Duesterberg PhD @vancanwin]

@LabLamba @KandoiSangeetha @_BrightFocus Super congrats @KandoiSangeetha

[Van Duesterberg PhD @vancanwin]

@drkeithsiau I wish I had these type of doctors. Thanks for sharing.

[alison greenberg @ALiS0NLAURA]

My texts this morning with a LEGAL abortion provider and nurse practitioner in Oregon.

We are in deep shit. Women will die.
#ImpeachClarenceThomas #Abortion #7weeks

[alison greenberg @ALiS0NLAURA]

HIRING ALERT:
@ruthhealthco is interviewing for a *paid* SOCIAL MEDIA INTERN role this summer! Undergrad, grad school, or if not schooling, at similar level of experience.

DM or reply to apply!
Share a sentence or two about why you're awesome + want to work in women's health.

[Polly Fordyce @fordycelab]

Have you ever wondered what the rest of a transcription factor (TF) beyond the DNA binding domain does? @nicole_delrosso and @BintuLab just published a beautiful tour-de-force paper that reveals 307 new activation domains and 592 repression domains! (1/3)

[Van Duesterberg PhD @vancanwin]

@acgt01 @lizworthey @mattmight @euanashley @acedens @brkorf Please reach out to me via my website https://t.co/yf94gfgJ1S. Also there are clinical trials but it depends on their visual acuity and such. https://t.co/yxeQ4lECSJ

[Daniel S. Levine @dslevine]

.@samiahalzaidy @Passage_Bio discusses the rare lysosomal storage disorder GM1 gangliosidosis, the company’s experimental therapy to treat the condition, and what’s known about the therapy from work that’s been done to date. @GlobalGenes #RARECast

https://t.co/9sUVhAdKIh

[David R. Liu @davidrliu]

Today we report in @ScienceMagazine a one-time base editing treatment for Spinal Muscular Atrophy (SMA), the leading genetic cause of infant mortality worldwide, affecting ~1 in 10,000 births. https://t.co/oLp8VN0Iiw
PDF: https://t.co/JoKm9BxW89
1/17

[Ethan Perlstein 1-to-N @eperlste]

Each new cure odyssey we launch with a pioneer family fuels my conviction that the world needs a Y Combinator for rare diseases.

@PerlaraPBC's investment thesis will be simple:

Fund entrepreneurial families to develop medicines for their kids
https://t.co/JTSLEzvgEI

[Ethan Perlstein 1-to-N @eperlste]

MedTwitter, I need your help:

I need a referrral for a pediatric hepatologist with expertise in non-cirrhotic hepatic encephalopathy and experience with diagnosing unsolved rare genetic disease cases involving the liver.

Please connect them to me ASAP!

[Stephanie Telesca @sn_telesca]

Thankful for @PerlaraPBC and all those working with us to find a cure. Because of them, I have #hope and I see a #bright future for E and all those with #RareDisease.

[✶ ela ✶ @elamadej]

Email we sent today to @fiftyyears portfolio founders about the SVB situation.

Sharing here so it can help others. Comments in the doc encouraged. https://t.co/yOsfWB7TSP