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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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 nit -
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 nit -
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 nit -
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 nit
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This week marks an important clinical milestone: first patient first dose with epalrestat in the treatment of
