Mutations in the RPGR gene are the most common cause of X-linked retinopathy, a hereditary retinal disease. We have thoroughly characterized the clinical features and natural history of a large cohort of patients with RPGR-associated X-linked retinopathy, documenting the progression from onset to blindness. This detailed analysis provides valuable insights into the disease’s timeline, offering crucial reference information for determining the optimal timing for ongoing RPGR-related gene therapy. Our findings aim to enhance the effectiveness of gene therapy treatments and improve patient outcomes by informing treatment decisions with a comprehensive understanding of disease progression. (By Jiawen Wu and Jihong Wu, https://jmg.bmj.com/content/61/10/973 )