Here is the three step formula for developing a novel therapy for Wolfram syndrome.
1. Understand the disease
2. Identify drug targets and biomarkers
3. Develop drugs and treat patients
Our patient registry and Wolfram clinic help us understand the disease. The second step depends on research using biotechnology in our lab. For the third step, we need to choose clinical endpoints. We also need help from the patient organization for the third step (i.e., clinical trial). My goal is to start a trial in 2014.