Month

December 2014

Wonderful people

So many people help us develop new treatment options for Wolfram syndrome and bring these to our patients. They look at our data, suggest the... Read More

Harvard Stem Cell Institute and CIRM

These two websites provide us with useful information regarding the stem cell-based therapies and clinical trials. Our institution is trying to catch up. http://hsci.harvard.edu/clinical-trials http://www.cirm.ca.gov/our-progress I... Read More

IND

IND stands for Investigational New Drug Application. To bring our new drug to our patients, I need to submit an IND to the FDA. We... Read More

Screening

We need multiple breakthroughs to achieve a cure for Wolfram syndrome. Based on my experience, “screening” often leads to a breakthrough. What do I mean... Read More