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Researchers have developed a highly efficient gene editing therapy that could potentially treat Stargardt disease, the most common form of inherited macular degeneration. This groundbreaking study, published in Nature Medicine on 8 January 2025, demonstrates the therapeutic use of a precision gene editing technology called base editing.
Gildeuretinol is an investigational new drug being developed by Alkeus Pharmaceuticals for the treatment of retinal diseases, particularly Stargardt disease and geographic atrophy secondary to age-related macular degeneration (AMD).
Following the successful clinical trials in LCA, researchers have been developing similar treatments using adeno-associated virus for age-related macular degeneration (AMD). To date, efforts have focused on long-term delivery of VEGF inhibitors to treat the wet form of macular degeneration. Whereas wet AMD is currently treated using frequent ...
WATERTOWN, Mass., Oct. 24, 2024 (GLOBE NEWSWIRE) -- EyePoint Pharmaceuticals, Inc. (NASDAQ: EYPT), a company committed to developing and commercializing innovative therapeutics to improve the lives of patients with serious retinal diseases, today announced that the first patient has been dosed in the Phase 3 LUGANO clinical trial of DURAVYU ...
To treat geographic atrophy secondary to age-related macular degeneration [4] Cipaglucosidase alfa: To treat late-onset Pompe disease with miglustat [4] Dabrafenib: Dostarlimab: Eflornithine: Elranatamab: To treat relapsed or refractory multiple myeloma after at least four lines of therapy [4] Enfortumab vedotin: Iptacopan
Macular degeneration treatment is safe and effective. The FDA approval was based on a review of scientific evidence showing that both drugs are “highly similar” to Regeneron Pharmaceuticals’ ...
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