Dr. Francis Collins is a world-famous medical researcher and my role model. He is a god in genetic medicine. He has been working on multiple rare diseases. Currently he is director of the National Institutes of Health. I often read one of his blogs on Sam Berns, his friend and patient with a rare disease.
I was inspired by this and wrote a blog on one of our patients, Ms. K.
I feel the same for our patients with Wolfram syndrome. Many of them are my friends and I would like to help them. We found a few candidate drugs that can theoretically delay the progression of the disease. I hope we can bring these drugs to our patients soon. We also need to develop gene therapy and regenerative therapy to provide a cure. I believe in the power of genetic medicine. I need more help and funds, but I am committed to make these happen.
Thank you for reading this blog. I am sending kind thoughts toward you.