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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Type A
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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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Cori Disease

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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Leigh Syndrome

"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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Mitochondria 2000x1000px
APOL1 kidney disease

"Genetic risk for APOL1 kidney
disease affects ~100 million
people worldwide"

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kidney disease
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Welcome to the archival website of Perlara 1.

Take a look around at the foundations of Perlara’s exciting new Guided Cures™ Journey.

Perlara scientists are working with highly motivated families, patient organizations and researchers to develop cures for rare diseases and learn how genes work across diseases to map connections to common ailments. We call these exciting journeys of discovery PerlQuests™

Organic Intelligence (OI): more cures for more individuals and diseases