Dr. Urano's Blog

Small Molecule

A small molecule for Wolfram?

We are looking for small molecules for developing novel therapeutics for Wolfram syndrome. What is a small molecule?

A small molecule is a low molecular weight chemical compound that may help regulate a biological process. So a small molecule almost means a drug. Some drugs are not small molecules. We have several small molecules that can potentially delay the progression of Wolfram syndrome. Three of them are FDA-approved small molecules, and one of them is a FDA-approved peptide.

I hope you will have a wonderful weekend!