If we had...We have...

I think about what I can do for patients with Wolfram syndrome every day. A cure for Wolfram can lead to a cure for diabetes, I believe.
http://wolframsyndrome.blogspot.com/2014/09/if-we-had.html

I am visualizing the world with:
1. Compassionate physicians who know how to manage the conditions.
2. Drugs that can stop the progression.
3. Therapies replacing the damaged tissues.

Thank you for your continuous support and encouragement. Thank you, Thank you, Thank you.

If we had...

Base on emails and phone calls from patients and their families, I am focusing my energy on the following three things. If we had these three, what would happen to us? Am I in the right direction?

1. Compassionate physicians who know how to manage the conditions.
2. Drugs that can stop the progression.
3. Therapies replacing the damaged tissues.

Thank you again for reading this. I am sending kind thoughts toward you.

Keep on moving forward

I thought a lot about how to create HOPE for patients suffering from Wolfram and other rare conditions during the Labor day weekend. If I were a patient, what would I want? These are the three things that I came up with.

1. Compassionate physicians who know how to manage the conditions.
2. Drugs that can stop the progression.
3. Therapies replacing the damaged tissues.

I think we have #1. Our friend, Dr. Bess Marshall, and her colleagues at the St. Louis Children's Hospital know a lot about Wolfram now. Our friend, Dr. Tammy Hershey, organized a research clinic, which helped these physicians learn about Wolfram syndrome. We need more physicians in different states and countries. 

How about #2 and #3? I feel that #2 is getting closer. #3 requires the development of regenerative medicine center. I am working on both.

Thank you for reading this. I hope you will have a wonderful day.

END WOLFRAM, END DIABETES, END ALS

I was watching the video that our friend and patient, Alejandro, was doing an ice bucket challenge. This project originally started for ALS. So I was checking the ALS-related websites and found the following youtube video. As most of you know, ALS (amyotrophic lateral sclerosis) is a neurodegenerative disease, leading to the progressive degeneration of the motor neurons. As a result, patients with ALS may become totally paralyzed. 

In these video series, many friends of Mr. Hiro Fujita, who has lost his voice due to ALS, read his messages. The project is called "END ALS." He was in the advertising business. So many celebrities read his messages. One of his messages was toward his mother. I would probably say the same word. There is an English caption in each video. He said, "Mom, Thank you."
https://www.youtube.com/watch?v=Dbnn4GqrgPc

He has a website, END ALS. He often uses the word, "HOPE." What do we need to help our patients suffering from life-threatening diseases? How can we create HOPE? Let's think about the basics.
1. Make correct diagnosis and refer them to the best specialists.
2. Develop therapeutics to stop the progression.
3. Replace damaged tissues.

These three should happen simultaneously. We need all of these. I want to END WOLFRAM, END DIABETES, END ALS, and...

I hope you will have a wonderful day. Thank you for reading this blog. Thank you for your supports. Thank you, thank you, thank you.

Thank you again, and Regenerate to Beat Degeneration.

I was surprised and glad to see that our patient and friend, Alejandro, was doing an ice bucket challenge for Wolfram syndrome. Thank you again, Ale and the team Alejandro!

I often get questions about "regenerative medicine." I really like the following video created by the Mayo Clinic. "Regenerate" is the opposite of "degenerate." Because Wolfram syndrome is a degenerative disease, the best way to counteract is to "regenerate" damaged tissues.
https://www.youtube.com/watch?v=qRCxvNXGx9g
http://www.mayo.edu/research/faculty/nelson-timothy-j-m-d-ph-d/bio-00027362

I hope you will have a wonderful Labor day today.