Thank you everyone for the Wolfram Research Clinic and Conference

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

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

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

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

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

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 […]

CURE4WOLFRAM 4.0

CURE4WOLFRAM 4.0
Dear Friends, Thank you so much for “coming to see me” today. It is always my pleasure and privilege to see you and talk to you. I feel your support and encouragement, and I am grateful for everything you have done for me, patients, patient organizations, the Snow Foundation, the Ellie White Foundation, Unravel Wolfram […]

Raise awareness

Raise awareness
I have three goals always on my mind: Raise Awareness, Improve Clinical Care, and Provide Novel Treatments for patients with Wolfram syndrome. As the Rare Disease Day is approaching, I would like to invite you to think about more about rare diseases, including Wolfram syndrome. There are 7,000 rare diseases and disorders that when combined […]

January 2018 updates

January 2018 updates
I would like to begin by thanking all of you for your continued support and encouragement. I appreciate it immensely. I am so grateful to have such supportive colleagues all over the world. Our drug-repurposing clinical trial of dantrolene sodium is ongoing. The trial began in January 2017. Twenty-two patients with Wolfram syndrome enrolled. Two […]

2018 goals

2018 goals
I thought a lot about our therapeutic development for Wolfram syndrome in the past several weeks. I feel that drug-repurposing may not be good enough. I firmly believe that we need a breakthrough therapy for Wolfram syndrome. This year, I would like to spend more time for developing regenerative gene therapy, especially for visual impairment. […]