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Prikvačeni tweet
This week marks an important clinical milestone: first patient first dose with epalrestat in the treatment of #PMM2-CDG! Here’s how we got here over the past 12 months.pic.twitter.com/V9AY0SYYHy
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Perlara proslijedio/la je Tweet
In many ways
@PerlaraPBC led the way in patient driven research models. It definitely emboldened many of us out there to push more.Hvala. Twitter će to iskoristiti za poboljšanje vaše vremenske crte. PoništiPoništi -
Perlara proslijedio/la je Tweet
Amazing progress for ultra-
#raredisease. We are grateful to@PerlaraPBC@eperlste for including us in this adventure.#OpenAccess FTW!https://twitter.com/perlarapbc/status/1223335171052560384 …Hvala. Twitter će to iskoristiti za poboljšanje vaše vremenske crte. PoništiPoništi -
Perlara proslijedio/la je Tweet
This week marks an important clinical milestone: first patient first dose with epalrestat in the treatment of #PMM2-CDG! Here’s how we got here over the past 12 months.pic.twitter.com/V9AY0SYYHy
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Perlara proslijedio/la je Tweet
Perlara demonstrates how personalized precision medicine can start with a single patient!https://twitter.com/PerlaraPBC/status/1223335171052560384 …
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Perlara proslijedio/la je Tweet
Road to a single patient IND - from scientific finding to first dose in patient. From our friends at
@PerlaraPBChttps://twitter.com/PerlaraPBC/status/1223335166543712256 …
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Perlara proslijedio/la je Tweet
From yeast to a patient in a year!https://twitter.com/PerlaraPBC/status/1223335166543712256 …
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Stay tuned for
#Nof1#PMM2-CDG trial updates in the weeks and months ahead! This work will be presented next month at the@SBPdiscovery#CDG conference in San Diego https://www.sbpdiscovery.org/calendar/2020-sbp-rare-disease-day-symposium …Prikaži ovu nitHvala. Twitter će to iskoristiti za poboljšanje vaše vremenske crte. PoništiPoništi -
November 2019: Published epalrestat story in
@DMM_Journal (https://dmm.biologists.org/content/12/11/dmm040584 …) December 2019: Epalrestat received by hospital outpatient research pharmacy Early January 2020: 50 mg tablets re-encapsulated into 5 mg tablets January 28th 2020: first dose!Prikaži ovu nitHvala. Twitter će to iskoristiti za poboljšanje vaše vremenske crte. PoništiPoništi -
April to July 2019: worked with FDA consultant to submit a 300+ page spIND package. August 2019: Study May Proceed letter sent by
@US_FDA September to November 2019: hospital IRB approval process November 2019: Engaged@Worldcourier to import epalrestat from JapanPrikaži ovu nitHvala. Twitter će to iskoristiti za poboljšanje vaše vremenske crte. PoništiPoništi -
February 2019: decision made by family and clinician to proceed with a single patient IND for epalrestat. March 2019: introduced to FDA regulatory consultant Caron & Associates (http://www.BioMDg.com ) by
@WylderNation, who had success getting a single patient IND cleared.Prikaži ovu nitHvala. Twitter će to iskoristiti za poboljšanje vaše vremenske crte. PoništiPoništi -
Sept 2018 to Jan 2019: based on initial discovery in yeast (https://www.g3journal.org/content/9/2/413 ) we hypothesized that aldose reductase inhibition would boost PMM2 enzymatic activity. So we tested all commercially available ARIs and discovered epalrestat as a first-in-class PMM2 activator.
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Perlara proslijedio/la je Tweet
Raghav lifts his head FOR FIRST TIME IN LIFE


Zero progress in 15months, 48hrs after a mild dose of N-Acetylcysteine this happens
I will make you stand on your own feet real soon Raghav
#CureGPX4#DrugRepurposingpic.twitter.com/LAXDYl2RIn
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Perlara proslijedio/la je Tweet
We're at 2600 signatures to send the message that taxpayers deserve access to the research they fund!! This one policy can save lives *and* unlock American innovation. Please keep spreading this to your networks!https://twitter.com/mattmight/status/1219846886984749057 …
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Perlara proslijedio/la je Tweet
By taking samples over time, we can look for how drug sensitivity changes to help design therapies and combinations that are much more effective than the standard treatment- Matt De Silva
#NotableANSWer https://bit.ly/2TNsgJD@ConorHale@FierceBiotechHvala. Twitter će to iskoristiti za poboljšanje vaše vremenske crte. PoništiPoništi -
Perlara proslijedio/la je Tweet
Patients and scientists: THIS IS NOT A DRILL. The White House is considering requiring open access to all taxpayer-funded research. Please sign to let The White House know that you strongly support this transformational initiative: http://www.oaintheusa.com/
#OAintheUSAHvala. Twitter će to iskoristiti za poboljšanje vaše vremenske crte. PoništiPoništi -
Perlara proslijedio/la je Tweet
Delighted to see this finally out after many years of work by Freeze lab!https://twitter.com/BWJones/status/1217805751533457409 …
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Perlara proslijedio/la je Tweet
Our latest publication is live at
@GeneticsGSA. "Cross-Species Complementation of Nonessential Yeast Genes Establishes Platforms for Testing Inhibitors of Human Proteins" Congratulations Akil.https://www.genetics.org/content/early/2020/01/14/genetics.119.302971 …Hvala. Twitter će to iskoristiti za poboljšanje vaše vremenske crte. PoništiPoništi -
Perlara proslijedio/la je Tweet
Published mitochondrial disease translational work using yeast by
@eperlste and team@PerlaraPBChttps://twitter.com/rhodophila/status/1215757940231147520 …Hvala. Twitter će to iskoristiti za poboljšanje vaše vremenske crte. PoništiPoništi -
Perlara proslijedio/la je Tweet
A really interesting history about biotechs, fundraising and VCs! A must read!
@eperlste explaining@PerlaraPBC's series Bhttps://twitter.com/eperlste/status/1212046919716855808 …
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