Happy New Year to all my friends around the world. I thought about what we need to accomplish to achieve a cure for Wolfram syndrome. Here are my goals in 2015.
1. Establish more Wolfram syndrome clinics
To improve the quality of life of our patients and know more about the disease, we need to establish more Wolfram clinics in the US and around the world. My goal is to help establish TWO MORE CLINICS IN THE US and TWO MORE CLINICS AROUND THE WORLD. We also need to establish clinics for adult patients who have different needs.
2. Know more about Wolfram syndrome
In 2014, I could not answer some of the questions from patients and their physicians. We need to know more about the disease. We need know more about dysphagia, apnea, general malaise, and washed-out feeling in Wolfram syndrome.
3. Conduct a clinical trial
This was one of my goals in 2014, but we could not accomplish it because of several reasons. We should complete preclinical and dose-escalation studies and make this happen.
4. Identify more drug targets and drugs
Last year, we could finally identify one drug target and a few drugs. We need to expand our pipelines. My goal is to identify TWO MORE TARGETS and TWO MORE CHEMICAL COMPOUNDS. A chemical compound that can activate MANF signaling would be needed.
5. Establish a platform for implementing regenerative therapies
This is an ongoing effort and I am trying to recruit surgeons and regulatory people for this.
6. Expand our network of patients with Wolfram syndrome
This is important for raising awareness of Wolfram syndrome. One of the major achievements last year was to establish a partnership with Wolfram syndrome UK. I will keep on giving lectures on Wolfram syndrome around the world and connecting with patients and patient organizations.
Let’s accomplish these together. I need your support and help.
Thank you fore reading this. I am quite hopeful.
Take care,
Fumi Urano