Perlara

@PerlaraPBC

Perlara PBC was founded on Feb 26, 2014 as the first therapeutics platform company with a mission to find cures for genetic diseases.

Berkeley, CA
Vrijeme pridruživanja: studeni 2013.

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  1. Prikvačeni tweet
    31. sij

    🚨 💊 🏥 This week marks an important clinical milestone: first patient first dose with epalrestat in the treatment of -CDG! Here’s how we got here over the past 12 months.

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  2. proslijedio/la je Tweet
    3. velj
    Odgovor korisnicima

    In many ways led the way in patient driven research models. It definitely emboldened many of us out there to push more.

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  3. proslijedio/la je Tweet
    3. velj

    Amazing progress for ultra-. We are grateful to for including us in this adventure. FTW!

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  4. proslijedio/la je Tweet
    31. sij

    🚨 💊 🏥 This week marks an important clinical milestone: first patient first dose with epalrestat in the treatment of -CDG! Here’s how we got here over the past 12 months.

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  5. proslijedio/la je Tweet
    1. velj

    Perlara demonstrates how personalized precision medicine can start with a single patient!

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  6. proslijedio/la je Tweet
    31. sij

    Road to a single patient IND - from scientific finding to first dose in patient. From our friends at

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  7. proslijedio/la je Tweet
    31. sij
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  8. 31. sij

    Stay tuned for -CDG trial updates in the weeks and months ahead! This work will be presented next month at the conference in San Diego

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  9. 31. sij

    November 2019: Published epalrestat story in () 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!

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  10. 31. sij

    April to July 2019: worked with FDA consultant to submit a 300+ page spIND package. August 2019: Study May Proceed letter sent by September to November 2019: hospital IRB approval process November 2019: Engaged to import epalrestat from Japan

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  11. 31. sij

    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 () by , who had success getting a single patient IND cleared.

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  12. 31. sij

    Sept 2018 to Jan 2019: based on initial discovery in yeast () 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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  13. proslijedio/la je Tweet
    29. sij

    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 ❤️

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  14. proslijedio/la je Tweet
    24. sij

    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!

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  15. proslijedio/la je Tweet
    22. sij

    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  

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  16. proslijedio/la je Tweet
    22. sij

    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:

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  17. proslijedio/la je Tweet
    16. sij

    Delighted to see this finally out after many years of work by Freeze lab!

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  18. proslijedio/la je Tweet
    14. sij

    Our latest publication is live at . "Cross-Species Complementation of Nonessential Yeast Genes Establishes Platforms for Testing Inhibitors of Human Proteins" Congratulations Akil.

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  19. proslijedio/la je Tweet
    14. sij

    Published mitochondrial disease translational work using yeast by and team

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  20. proslijedio/la je Tweet
    14. sij

    A really interesting history about biotechs, fundraising and VCs! A must read! explaining 's series B

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