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Endpoints and Design for Clinical Trials in USH2A-Related Retinal Degeneration: Results and Recommendations From the RUSH2A Natural History Study.
Transl Vis Sci Technol
October 2024
Jaeb Center for Health Research, Tampa, FL, USA.
Article Synopsis
- The study aims to evaluate how functional and structural assessments can serve as endpoints in clinical trials for retinal degeneration linked to USH2A mutations.
- Participants with specific visual capabilities underwent various eye tests over four years, focusing on understanding changes in their vision.
- Findings indicated that certain tests were more sensitive to detecting changes, influencing the design of future clinical trials related to this condition.
Safety and efficacy of ATSN-101 in patients with Leber congenital amaurosis caused by biallelic mutations in GUCY2D: a phase 1/2, multicentre, open-label, unilateral dose escalation study.
Lancet
September 2024
Center for Hereditary Retinal Degenerations, Scheie Eye Institute, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA. Electronic address:
Background: Leber congenital amaurosis 1 (LCA1), caused by mutations in GUCY2D, is a rare inherited retinal disease that typically causes blindness in early childhood. The aim of this study was to evaluate the safety and preliminary efficacy of ascending doses of ATSN-101, a subretinal AAV5 gene therapy for LCA1.
Methods: 15 patients with genetically confirmed biallelic mutations in GUCY2D were included in this phase 1/2 study.
Improvement of oxidized myofibrillar protein gel properties by black rice extract.
Food Chem X
March 2024
School of Marine and Biological Engineering, Yancheng Teachers' University, Yancheng 224007, China.
In order to investigate the effects of black rice extract (BE) on the composition of oxidized myofibrillar protein (MP) gel, different concentrations of BE (0, 10, 20, 50 mg g) were analyzed experimentally. Results revealed that the addition of small doses of BE significantly inhibited the formation of carbonyl groups in oxidized MP, and improved surface hydrophobicity and gel water holding capacity. Additionally, 10 and 20 mg g BE increased the ordered structure of oxidized MP.
View Article and Find Full Text PDFIntroduction: AURIGA is the largest real-world study to date to evaluate intravitreal aflibercept (IVT-AFL) treatment of diabetic macular edema or macular edema secondary to retinal vein occlusion (RVO) in routine clinical practice. Here, we report the 24-month outcomes in the RVO cohort from France, Germany, Italy, and Taiwan.
Methods: AURIGA (NCT03161912) was a prospective observational study.
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