I appreciate the importance of basic research because it provides us with drug targets and new technologies. To achieve a cure for Wolfram and other rare diseases, it is important for us to translate research into clinical practice. For example, we have recently found a drug target and a potential drug for Wolfram syndrome. To bring this drug to our patients, we need to carry out dose-escalation studies and PK study, and design a clinical trial. We need to secure the intellectual property of our drug. I did not learn these during medical school, residency, or fellowship. Who are the experts? The answer is “medical officers” in the pharmaceutical industry. I am learning these now because it is important, and the Snow foundation has been helping me on this.
Thank you for reading this blog. I am doing my best. I understand that we are racing against time. I always hope that our patients’ conditions are stable.