# drug repurposing (blogs) — RSS Amplifier

Recent posts from the 1 feeds in the RSS Amplifier directory that cover drug repurposing.

Page: <https://rssamplifier.com/topics/drug-repurposing/blogs>  
Feed: <https://rssamplifier.com/topics/drug-repurposing/blogs.md>

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## [Restoring balance to the flux](https://perlara.substack.com/p/restoring-balance-to-the-flux)

_2025-07-29 · Ethan O. Perlstein, Ph.D. · Perlara’s Cure Odysseys_

CDGs are a 200+ gene family of inherited metabolic diseases, GMPPA-CDG and SRD5A3-CDG to name a few. Turns out GMPPA deficiency is rescued by SRD5A3 deficiency 🤯. Two genetic wrongs can make a right.

## [FBXO31 Cure Roadmap](https://perlara.substack.com/p/fbxo31-cure-roadmap)

_2025-01-13 · Ethan O. Perlstein, Ph.D. · Perlara’s Cure Odysseys_

The first step toward a cure is a treatment, and the first step toward a treatment is a research roadmap.

## [Community medicine](https://perlara.substack.com/p/community-medicine)

_2024-12-11 · Ethan O. Perlstein, Ph.D. · Perlara’s Cure Odysseys_

A rebel alliance of rare parents united as the PIGN-CDG Cure Collective to fund a drug repurposing project. One of the top 3 hits is ascorbyl palmitate aka fat-soluble vitamin C - and it's working!

## [All in the FAMily](https://perlara.substack.com/p/all-in-the-family)

_2024-10-26 · Ethan O. Perlstein, Ph.D. · Perlara’s Cure Odysseys_

An unbiased fly-powered drug repurposing screen for FAM177A1 deficiency turned up the generic ALS drug riluzole as the top hit and inspired a family-initiated, family-led pioneer N-of-1 study.

## [Fuel It Still](https://perlara.substack.com/p/fuel-it-still)

_2024-10-16 · Ethan O. Perlstein, Ph.D. · Perlara’s Cure Odysseys_

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 yielding results after 12 weeks of NMN treatment.

## [Repurposing is a gateway drug](https://perlara.substack.com/p/repurposing-is-a-gateway-drug)

_2024-10-06 · Ethan O. Perlstein, Ph.D. · Perlara’s Cure Odysseys_

A drug repurposing project for the ultra-rare mitochondrial disease MEPAN has yielded a clinical candidate and serendipitously illuminated a pathway to designer pharmacological chaperones using GenAI.