Perlara’s Cure Odysseys
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Pharma Chameleon
Often hiding in plain sight, a small molecule can have multiple pharmacological identities. Context-switching and shape-shifting pharmacophores keep…
Sep 20
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Perlara PBC
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Pharma Chameleon
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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…
Sep 9
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Perlara PBC
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ADSL Cure Odyssey
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August 2023
FAMily ties
Less than three months ago, FAM177A1 Research Foundation hosted its first research roundtable. Drug repurposing and biomarker discovery projects are now…
Aug 29
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Perlara PBC
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FAMily ties
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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…
Aug 3
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Perlara PBC
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PIGS-CDG Cure Odyssey
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July 2023
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…
Jul 26
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Perlara PBC
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PIGW-CDG Cure Odyssey
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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…
Jul 5
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Perlara PBC
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AFG3L2 Cure Odyssey
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June 2023
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…
Jun 29
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Perlara PBC
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GMPPA-CDG Cure Odyssey
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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…
Jun 12
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Perlara PBC
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PIGN Cure Collective
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May 2023
Yeast-powered drug repurposing screens for SURF1 Leigh Syndrome are done 💊♻️ ✅
On behalf of Cure Mito Foundation, we completed high-throughput drug screens and dose response studies in yeast. Next up: validating hits in SURF1…
May 26
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Perlara PBC
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Yeast-powered drug repurposing screens for SURF1 Leigh Syndrome are done 💊♻️ ✅
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CureGPT
Everyone's chattering these days about how AI will save or destroy humankind. Setting aside the hype and the noise, how can AI empower families and…
May 19
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Perlara PBC
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CureGPT
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Drug repurposing for PACS2 with Cell Painting
The CureMap we created for PACS2 Research Foundation recommended drug repurposing that combines patient fibroblasts and an unbiased image-based…
May 13
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Perlara PBC
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Drug repurposing for PACS2 with Cell Painting
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FAM177A1 Cure Odyssey
After her two kids completed a diagnostic odyssey with the UDN, Jill Hawkins created the FAM177A1 Research Foundation. The inaugural (virtual) FAM177A1…
May 11
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Perlara PBC
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FAM177A1 Cure Odyssey
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