Loading Curie Briefs...
Getting the latest healthcare news for you
Getting the latest healthcare news for you
A modified version of vitamin A could slow vision loss in Stargardt disease, a rare genetic eye condition. A 2-year clinical trial found that gildeuretinol acetate — a deuterium-enriched form of vitamin A — reduced the growth of retina-damaging lesions by over 20%, with no serious safety concerns. It's a promising step forward for a disease that currently has no approved treatments.
Stargardt disease is a rare inherited condition where a genetic mutation causes vitamin A to clump into toxic deposits in the retina, gradually destroying vision. Researchers tested whether swapping out the body's natural vitamin A with a modified, deuterium-enriched version — called gildeuretinol acetate — could slow that damage. The answer, from a 2-year trial across seven US clinics, appears to be yes.
The trial enrolled 50 patients aged 12 and older, split across different dosing groups. By the end of the study, patients on the drug saw retinal lesions grow at a rate of 0.182 mm/year, compared to 0.232 mm/year in untreated patients — a 21.6% relative reduction. Side effects were mild to moderate and comparable across all groups.
By the Numbers:
Why it matters: Stargardt disease has no approved treatments, and vision loss is currently considered irreversible. A therapy that meaningfully slows retinal deterioration — even without restoring sight — could be a game-changer for patients and families navigating this diagnosis.
