We identified tool compounds, repurposable drugs as well as novel chemotypes that rescue worm and fly disease models.
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Niemann-Pick Type A
We identified overlapping hits between worm and patient fibroblast drug repurposing screens.
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We identified natural products and repurposable drugs that rescue yeast and worm disease models.
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Multiple Sulfatase Deficiency
MSD mutant flies are in the lab and phenotyping has begun!
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"Perlara’s work will help put children with a GNAO1-related neurodevelopmental disorder further on the radar of researchers and pharmaceutical companies.”
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"The collaborative drug discovery effort gives
hope for new insights about CLS and better lives for our children.”
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We’re excited to break new ground
scientifically with a yeast-to-organoid drug screening pipeline for pancreatitis and cystic fibrosis.
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Perlara’s expertise will accelerate the identification of small molecules as potential treatments for GSD patients.
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With children losing ground to this disease every day we have to we are hopeful that our PerlQuests will bring us ever closer to help for patients with Sanfilippo syndrome.
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We are developing nematode and fly disease models and screening compounds for promising leads. Stay tuned.
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"A new treatment for Leigh syndrome could have broader implications for aging, sports medicine, traumatic brain injury, neurodegeneration and other indications."
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Perlara scientists are working with highly motivated families, patient organizations and researchers to develop cures for rare diseases and learn how genes work acrossdiseases to map connections to common ailments. We call these exciting journeys of discovery PerlQuests™