Rare diseases may affect only a few at a time, but together they impact 6–8% of the population, with 6000–8000 conditions identified globally and around 450 known in India. Many are genetic, often affecting children, yet diagnosis and treatment are delayed because of limited awareness, research gaps, high costs , and restricted access to quality care. Although the Government of India recognised rare diseases as a priority in 2017 through the National Policy for Rare Diseases, families still face enormous challenges in getting timely , affordable support. 1 This is where platforms like Rx4U matter. By helping families find verified assistance options, navigate documentation, and access available schemes or funding channels, Rx4U reduces confusion and bridge the gap between urgent need and actual access, making an overwhelming journey feel a little less heavy. The Challenge of Accessing Rare Disease Medicines in India ...