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A cure for Wolfram syndrome could lead to a cure for diabetes.

Friday, January 17, 2014

Three step formula for developing therapeutics and Three activities to execute it



1. Three-Step Formula For Developing Therapeutics for Wolfram syndrome

Here is my three-step formula for developing therapeutics for Wolfram syndrome.

Step 1. Understand the disease
Step 2. Identify drug targets and biomarkers
Step 3. Develop drugs and treat patients

To execute these three steps, we have three ongoing activities at the Washington University Medical Center.


2. Three Activities at Washington University Medical Center

2-1. Wolfram syndrome International Patient Registry
We are collecting the medical records, blood samples, and genetic information from patients with Wolfram syndrome through the internet. (Step 1).

2-2. Wolfram syndrome Clinic
We host a Wolfram syndrome clinic every year to understand the natural history of the disease. Many physicians and staff members have been involved in this activity. It is important to understand the major clinical problems and the progression of the disease (Step 1). It is also important to determine the baseline conditions of each patient for a future clinical trial.

2-3. Patient-based therapeutic development
We are taking an unconventional approach to develop therapeutics for Wolfram syndrome. I would call it "patient-based therapeutics." I discussed this concept yesterday.