Perlara’s Cure Odysseys

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October 2024

All in the FAMily
An unbiased fly-powered drug repurposing screen for FAM177A1 deficiency turned up the generic ALS drug riluzole as the top hit and inspired a…
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Ethan O. Perlstein, Ph.D.
Fuel It Still
Boosting levels of the metabolic fuel NAD+ rescues DHDDS deficiency in a yeast avatar. 3 pioneer N-of-1 studies are now underway. Rosie's N-of-1 is…
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Ethan O. Perlstein, Ph.D.
Repurposing is a gateway drug
A drug repurposing project for the ultra-rare mitochondrial disease MEPAN has yielded a clinical candidate and serendipitously illuminated a pathway to…
 • 
Ethan O. Perlstein, Ph.D.

June 2024

May 2024

April 2024

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…
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Ethan O. Perlstein, Ph.D.
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…
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Ethan O. Perlstein, Ph.D.
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…
 • 
Ethan O. Perlstein, Ph.D.

February 2024

January 2024

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