fenretinide and Stargardt-Disease

Stargardt disease, an inherited macular dystrophy caused by mutations in the ABCA4 gene encoding a retinal transporter protein, is the most prevalent form of macular degeneration in children. Patients with Stargardt disease develop severe vision loss within their first or second decades of life, which progresses to irreversible decreased visual acuity in almost all cases. Presently, there are no standard treatments for Stargardt disease. However, encouraging progress has been made in the development of innovative approaches to preventing vision loss in Stargardt patients.. Among the promising treatment candidates include ALK-001, fenretinide, and A1120 as pharmacological agents to modulate the visual cycle, StarGen

Topics: Antineoplastic Agents; Fenretinide; Genetic Therapy; Humans; Ligands; Macular Degeneration; Piperidines; Retinal Pigment Epithelium; Stargardt Disease; Stem Cell Transplantation