Two-birds-one-stone strategy shows promise in RNA-repeat expansion diseases

Excellent news! This sounds very promising!

"... A new strategy for treating a variety of diseases known as RNA-repeat expansion disorders, which affect millions of people, has shown promise in proof-of-principle tests conducted by scientists at Scripps Research. ...
The results suggest that someday, a handful of well-targeted drugs might be able to treat the more than 40 human disorders—including Huntington’s disease and variants of amyotrophic lateral sclerosis (ALS)—that arise from RNA-repeat expansions. ...
In the study, published in Cell Chemical Biology, the scientists showed that a potential drug molecule they developed can neutralize the toxic RNA that causes two distinct repeat-expansion disorders, myotonic dystrophy 1 (DM1) and Fuchs endothelial corneal dystrophy (FECD)."

"... we designed a lead small molecule that binds the structure of the r(CUG) repeat expansion [r(CUG) exp] that causes myotonic dystrophy type 1 (DM1) and Fuchs endothelial corneal dystrophy (FECD) and rescues disease biology in patient-derived cells and in vivo. Interestingly, the compound's downstream effects are different in the two diseases, owing to the location of the repeat expansion. ..."

Two-birds-one-stone strategy shows promise in RNA-repeat expansion diseases | Scripps Research A compound designed at Scripps Research works in early tests against myotonic dystrophy 1 and Fuchs endothelial corneal dystrophy, which together affect millions of people.

Here is the respective research paper:

A Small Molecule that Binds an RNA Repeat Expansion Stimulates Its Decay via the Exosome Complex