Perlara’s Cure Odysseys
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ECHS1 Cure Odyssey
Fibroblasts and flies joined forces in a multi-species drug repurposing campaign that yielded p38 MAPK inhibitors as top rescuers for this ultra-rare…
Apr 30
•
Ethan O. Perlstein, Ph.D.
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ECHS1 Cure Odyssey
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Two nutraceuticals are the top drug repurposing hits for ALG11-CDG
An initial pioneer ALG11-CDG family blazed a trail with a pilot 2,200-compound screen, paving the way for a second ALG11-CDG family to grab the baton…
Apr 25
•
Ethan O. Perlstein, Ph.D.
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Two nutraceuticals are the top drug repurposing hits for ALG11-CDG
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Biomarker discovery for FAM177A1
When a disease is both rare and new to medicine, the only recourse is research. FAM177A1 Research Fund is excited to shared preliminary results from a…
Apr 23
•
Ethan O. Perlstein, Ph.D.
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Biomarker discovery for FAM177A1
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FOXG1 Cure Odyssey
A mother’s “darkest days” leads to a foundation and a magazine dedicated to families caring for children with rare diseases.
Feb 29
•
Laura DeFrancesco
and
Ethan O. Perlstein, Ph.D.
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FOXG1 Cure Odyssey
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SURF1 ReFRAME recap
On behalf of Cure Mito Foundation, last week we completed a comprehensive 13,500-compound drug repurposing screen using a Leigh Syndrome yeast avatar of…
Feb 5
•
Ethan O. Perlstein, Ph.D.
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SURF1 ReFRAME recap
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SURF1 yeast hits validated in patient fibroblasts
Cure Mito Foundation partnered with Perlara on yeast-powered drug repurposing screens for SURF1 Leigh Syndrome. We found three compounds that increase…
Feb 5
•
Ethan O. Perlstein, Ph.D.
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SURF1 yeast hits validated in patient fibroblasts
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Drug repurposing with yeast avatars of SURF1 Leigh Syndrome
We started working with Cure Mito in August on a yeast drug repurposing project based on the template of our previous work with UCSF on MEPAN. Last week…
Feb 5
•
Ethan O. Perlstein, Ph.D.
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Drug repurposing with yeast avatars of SURF1 Leigh Syndrome
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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…
Feb 5
•
Ethan O. Perlstein, Ph.D.
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Yeast-powered drug repurposing screens for SURF1 Leigh Syndrome are done 💊♻️ ✅
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Perlara’s Cure Odysseys
Stories from pioneering families battling genetic diseases at the frontier of precision medicine.
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