To qualify for treatment with a personalized experimental ASO, each patient’s genetic mutation or change in gene function must be nano-rare meaning the same mutation is only found in approximately in 1 – 30 patients in the world.   Each patient must live in the United States and have a diagnosis and the genetic cause must be defined.   Patients must have a gene target in an organ that we target, and the patient must be cared for by a research physician and an institution (tertiary care center) experienced in managing Research INDs.