Real Progress in the Search for a Treatment

Progress. All rare disease parents want to see progress in the search for a treatment for their child. In the past two years, Sanath has worked diligently to make that progress happen and it is beginning to pay off. When we started Raising Rare, our goal was to follow the story of baby Raghav and his parents Sanath and Ramya. A second goal was to help other rare parents learn from their journey. One part of this journey is the process of scientific research. Two years ago, Sanath had no exposure to this process and even had to look up the definition of a gene. In this episode, he sounds like a seasoned researcher. He takes us from where they started, hoping to repurpose an existing drug, through the first few treatments they tried, and finally to the program they have established to look at the potential of thousands of medicines. It is a high-throughput screening program that will help them determine whether any of these medicines will help Raghav’s cells grow. We even discuss how artificial intelligence is transforming the way that these types of experiments can be run. Oh, how far he has come.