Goals for 2020: One Team
Dear Friends, Happy New Year. Thank you for your email, letters, and messages in the past few weeks. I appreciated your encouragement. I am determined to make 2020 the game-changing year for us. Three things are always on my mind: Improve clinical care, Raise awareness, and Provide a cutting-edge treatment for Wolfram syndrome. I have […]
Thank you for your support. Our progress in 2019.
Dear Friends, As the year 2019 is coming to an end, I would like to summarize our progress this year. Tomorrow, I would like to tell you my plan for 2020. We have been focusing our efforts on the development of regenerative and gene therapy. We successfully created the adeno-associated virus (AAV) that can deliver […]
Trial updates and gene therapy
Thank you for your continued support and encouragement. I would like to update you about our progress on the therapeutic development for Wolfram syndrome. Our clinical trial of dantrolene sodium for adult and pediatric patients with Wolfram syndrome has been going well. Based on the results of dantrolene trial, I feel confident that counteracting ER […]
Thank you, Wolfram UK!
I attended the Wolfram Syndrome UK annual conference on September 28, 2019. I started attending this important conference in 2014. Now I know many patients and families in the UK. This conference is special for me because I have a chance to discuss challenges patients and families have face to face. This year was also […]
Thank you everyone for the Wolfram Research Clinic and Conference
Dear Friends, This year’s Wolfram research clinic was successful again. The community conference hosted by the Snow Foundation was a big success too. I would like to thank all the participants, families, and staff for the research clinic and community conference. Please feel free to contact me if you have any questions regarding the genetic […]
On-going efforts and answering your questions
I have received many emails and phone calls regarding our therapeutic development for Wolfram syndrome in the past several weeks. Thank you so much for your kind words and encouragement. I would like to update you about our ongoing efforts on therapeutic development. I hope this blog will answerer some of your questions. A Drug-Repurposing […]
Regenerative gene therapy
Thank you so much for visiting our website for Wolfram syndrome patients, their families, friends, and supporters. I hope you and your family are enjoying the beautiful autumn season and nature. I love to do a nature walk especially in autumn, and I came up with the following idea during my nature walk. I’d like […]
Molecular prosthetics
Dear Friends, Thank you so much for your support. I hope you are enjoying this beautiful autumn season with your family, and it is time for me to update you about our progress. Today, I’d like to talk about one of the new drugs we are developing as we have made significant progress on this […]
Humanized Wolfram syndrome mice and rats
I’d like to begin by thanking all of you for your continued support, trust, and faith in me. I always feel your support and energy. It is always my pleasure to update you about our progress. To achieve a cure for Wolfram syndrome, we need breakthrough treatments. We currently have three new candidate drugs that […]
Wolfram syndrome workshop in Paris
It is nice to see you again. I always appreciate your support, trust, and faith in me. I see patients with Wolfram syndrome every week and receive phone calls and emails from patients and their families every single day. It is definitely urgent to slow the progression of the disease and develop novel treatments for […]
This close
Dear Friends, Nice to see you again. Thank you so much for following my mission & vision and being the kindest person. I think about patients with Wolfram syndrome and their families and friends every morning. That’s one of the first things I do every day at 4:30 am. I would like to know their […]
Underdiagnosed
Dear Friends, It is always nice to talk to you. Thank you so much for reading this. I firmly believe that Wolfram syndrome is an underdiagnosed disease. Wolfram syndrome is characterized by juvenile-onset diabetes, optic nerve atrophy, diabetes insipidus, deafness, neurogenic bladder, and symptoms related to brain cell dysfunction. However, I have discovered that Wolfram […]