Perlara’s Cure Odysseys

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ADSL Cure Odyssey
A scientist and mother from Belgrade reached out last year about therapeutic options for her daughter suffering from an ultra-rare disease called ADSL…
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Perlara PBC
FAMily ties
Less than three months ago, FAM177A1 Research Foundation hosted its first research roundtable. Drug repurposing and biomarker discovery projects are now…
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Perlara PBC
PIGS-CDG Cure Odyssey
We call them odysseys for good reason. The scientific method involves unexpected detours and cul-de-sacs. But persistence and patience in science pay…
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Perlara PBC
PIGW-CDG Cure Odyssey
When pigs fly, the saying goes. There's a group of congenital disorders of glycosylation known as the PIG's, and they've taken flight with yeast-powered…
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Perlara PBC
AFG3L2 Cure Odyssey
Rita and Daniel launched a yeast-powered drug repurposing project on behalf of their son Milo, who has two pathogenic variants in his AFG3L2 gene. The…
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Perlara PBC
GMPPA-CDG Cure Odyssey
Using worm and fly avatars, we identified three hits with a shared pharmacophore that appears to target another CDG gene. We're testing a metabolic…
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Perlara PBC
PIGN Cure Collective
PIGN-CDG families are funding yeast-powered drug repurposing screens and a fibroblast biobank for hit validation. We just completed a pilot repurposing…
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Perlara PBC
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Perlara’s Cure Odysseys
Stories from pioneering families battling genetic diseases at the frontier of precision medicine.

Perlara’s Cure Odysseys

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