Perlara’s Cure Odysseys
Subscribe
Sign in
Home
Podcast
Archive
About
Latest
Top
Discussions
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.
1
Share this post
ECHS1 Cure Odyssey
perlara.substack.com
Copy link
Facebook
Email
Note
Other
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.
Share this post
Two nutraceuticals are the top drug repurposing hits for ALG11-CDG
perlara.substack.com
Copy link
Facebook
Email
Note
Other
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.
Share this post
Biomarker discovery for FAM177A1
perlara.substack.com
Copy link
Facebook
Email
Note
Other
February 2024
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.
2
Share this post
FOXG1 Cure Odyssey
perlara.substack.com
Copy link
Facebook
Email
Note
Other
1
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.
Share this post
SURF1 ReFRAME recap
perlara.substack.com
Copy link
Facebook
Email
Note
Other
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.
Share this post
SURF1 yeast hits validated in patient fibroblasts
perlara.substack.com
Copy link
Facebook
Email
Note
Other
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.
Share this post
Drug repurposing with yeast avatars of SURF1 Leigh Syndrome
perlara.substack.com
Copy link
Facebook
Email
Note
Other
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.
Share this post
Yeast-powered drug repurposing screens for SURF1 Leigh Syndrome are done 💊♻️ ✅
perlara.substack.com
Copy link
Facebook
Email
Note
Other
Leigh Syndrome drug repurposing updates
In collaboration with Cure Mito Foundation and using a SURF1-deficient yeast model, we're validating hits from the pilot screen, and we just screened a…
Feb 5
•
Ethan O. Perlstein, Ph.D.
Share this post
Leigh Syndrome drug repurposing updates
perlara.substack.com
Copy link
Facebook
Email
Note
Other
January 2024
Unexpected PLOD1 twist
Collagen is a slippery eel. After trying and failing to visualize collagen inside PLOD1-kEDS fibroblasts, we pivoted to measuring collagen secreted by…
Jan 31
•
Ethan O. Perlstein, Ph.D.
Share this post
Unexpected PLOD1 twist
perlara.substack.com
Copy link
Facebook
Email
Note
Other
The Anti-Portfolio of Pharma
The unlikely intersection of overlooked diseases, overlooked assets and overlooked founders is the place we've called home for the past 10 years. When…
Jan 7
•
Ethan O. Perlstein, Ph.D.
8
Share this post
The Anti-Portfolio of Pharma
perlara.substack.com
Copy link
Facebook
Email
Note
Other
4
December 2023
DHDDS Cure Odyssey
Two pioneer families created an Anglo-American alliance and pooled resources in order to fund a yeast-powered drug repurposing project for DHDDS…
Dec 27, 2023
•
Laura DeFrancesco
and
Ethan O. Perlstein, Ph.D.
4
Share this post
DHDDS Cure Odyssey
perlara.substack.com
Copy link
Facebook
Email
Note
Other
Share
Copy link
Facebook
Email
Note
Other
This site requires JavaScript to run correctly. Please
turn on JavaScript
or unblock scripts