Passionate eye doctors

We are developing a treatment for optic nerve dysfunction and death in Wolfram syndrome, and creating different types of eye cells from iPS cells derived from patients' skin cells. I feel fortunate that I could find eye doctors who are willing to work closely with my team.

I hope you had a wonderful July 4th!

Inhaled insulin

Inhaled insulin has been approved by the FDA, and I received many questions about "inhaled insulin" this week. I recommend that you consult with your physician about the use of inhaled insulin because each patient is different.

Because it is inhaled, it is quickly absorbed from the cells in the lungs. So its levels increase quickly in the blood. Thus, we probably need less insulin to get the same effects, which may be beneficial for patients. Importantly, we don't need needles.

Time will tell if this is a game changer or not. I always feel that we should keep on trying to make a better drug for patients.

Need to know about genetic testing

Although I believe in the power of genetic testing to make a correct diagnosis and create a treatment plan, I acknowledge that there are limitations to it. Genetic testing gives us important information to understand what's going on in each patient, whereas it does not perfectly predict what will happen to the patient at hand. I feel that genetic testing is a tool to choose an appropriate treatment.
http://wolframsyndrome.dom.wustl.edu/

A Three-Step Formula 2.0 for Providing a Cure for Wolfram Syndrome

Here is my new strategy. We are working on all of these steps in parallel.

Each patient is different

As we have learned more about patients with Wolfram syndrome, we have discovered that each patient is different. Dr. Timothy "Tim" Barrett, a world-famous expert of Wolfram syndrome in England, told me the same thing. So what should we do? I think that we should treat each patient differently. We should have multiple options for Wolfram syndrome. I am moving into this direction, and making progress.