A Simple Vitamin May Hold the Key to Treating Rare Genetic Diseases

Researchers used a genetics-first screen to match vitamins to rare diseases with specific mutations.

In mice with lethal NAXD deficiency, high-dose vitamin B3 greatly improved survival and reduced disease symptoms.

The study also pointed to other genetic disorders that may respond to vitamin B2 or other targeted supplementation.

The findings suggest some rare diseases could be treated with low-cost, widely available vitamins when the mutation is known.