Physics:Gildeuretinol
From HandWiki
Gildeuretinol is an investigational new drug being developed by Alkeus Pharmaceuticals, Inc. for the treatment of retinal diseases, particularly Stargardt disease and geographic atrophy secondary to age-related macular degeneration (AMD). Stargardt disease is caused by a defect in the ABCA4 gene that clears toxic byproducts resulting from the dimerization of vitamin A. Gildeuretinol is new molecular entity designed to reduce the dimerization of vitamin A in the eye without affecting the visual cycle.[1]
Gildeuretinol has received breakthrough therapy, orphan drug and Pediatric Rare Disease designations from the U.S. Food and Drug Administration.[2]
References
- ↑ "The ABCs of Stargardt disease: the latest advances in precision medicine". Cell & Bioscience 14 (1). July 2024. doi:10.1186/s13578-024-01272-y. PMID 39060921.
- ↑ "Gildeuretinol for Stargardt disease receives Rare Pediatric Disease, Fast Track Designations". Contemporary Pediatrics. 22 November 2024. https://www.contemporarypediatrics.com/view/gildeuretinol-stargardt-disease-rare-pediatric-disease-fast-track-designations.
