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.
