Search results
17 cze 2022 · A new drug to treat Stargardt disease has been granted a fast-track review by the Food and Drug Administration in the US, to help make it available to patients quicker.
9 lis 2023 · The drug is the first and only medicine to treat Stargardt disease by targeting the underlying causes of the disease and reducing the rate of vitamin A dimerization. Stargardt disease is an inherited form of macular degeneration with no FDA-approved treatment available to date.
19 paź 2024 · Key Takeaways. Tinlarebant reduces vitamin A delivery to the eye, slowing bisretinoid formation in Stargardt disease, caused by ABCA4 gene mutations. Phase two study showed an 80% reduction in plasma RBP4 and decreased visual acuity loss in adolescents with Stargardt disease.
29 sty 2024 · Another drug to potentially treat Stargardt has shown positive results in slowing down the progression of the disease in a recent trial. Alkeus Pharmaceuticals has revealed promising early results from its TEASE-3 clinical trial of the drug ALK-001.
24 paź 2024 · says. Ascidian, which is named for a group of marine organisms in which trans splicing is rampant, launched its clinical trial this summer to test whether this mechanism can slow progression of Stargardt disease, an inherited version of macular degeneration caused by defects in the ABCA4 gene, whose protein helps protect the eye from toxins.
2 dni temu · Christopher Cioffi, PhD, Thomas and Constance D’Ambra Professor in Organic Chemistry at Rensselaer Polytechnic Institute, is collaborating with Konstantin Petrukhin, PhD, Professor of Ophthalmic Science at Columbia University, to develop compounds to treat dry age-related macular degeneration (AMD) and Stargardt disease.. Their research has led to the identification of an advanced ...
26 lip 2024 · Drug therapy. Stem cell therapy. Stargardt disease (STGD) is the most common form of inherited juvenile macular dystrophy and is caused by sequence variants in the ABCA4 gene. Due to its genetic complexity and phenotypic variability, STGD poses significant therapeutic challenges.
