Updates on Therapeutic Development
February 6, 2021 Dear Friends, I wish and pray that 2021 is a year of splendid health and big success for you. Despite all the uncertainty, troubles, and challenges on our planet, I have focused my mind and efforts on helping and saving our patients with Wolfram syndrome and WFS1-related disorders. I have been adhering […]
Therapeutic Development Pipelines and Timeline
Dear Friends, First of all, I want to express my gratitude for everything you’ve helped me achieve here. Your kind words and encouragement keep me motivated and inspired. My three guiding principles are: Improve clinical care, Raise awareness, and Provide a cutting-edge treatment for Wolfram syndrome. Here is our progress: A Drug-Repurposing Clinical Trial Our […]
Continue working as one team
Dear Friends, Thank you for visiting our Wolfram Syndrome website. I hope you and your family are safe, strong, and healthy on a brighter future. I have no doubt better days are ahead, and we can rise about this crisis. Many of you have asked me if I need to assist with COVID-19 research or […]
My deepest and sincere gratitude to you
Dear Friends, Today, I would like to show my gratitude to you. I have received hundreds of emails in the past few days regarding our recent advances in regenerative gene therapy for Wolfram Syndrome that could lead to breakthrough therapy not only for Wolfram Syndrome but also for retinal degeneration, diabetes, and neurodegenerative disorders. All […]
Toward personalized regenerative gene therapy for Wolfram syndrome
Dear Friends, I hope you and your family are safe and well during this COVID-19 pandemic. Three things are always on my mind: Improve clinical care, Raise awareness, and Provide a cutting-edge treatment for Wolfram syndrome. As I mentioned on January 1st, I am determined to make 2020 the game-changing year for us despite this […]
Rare Disease Day 2020
Dear Friends, Happy Rare Disease Day. Thank you for your emails and messages in the past few days. I appreciate you shared your feelings with me and encouraged me regarding our therapeutic development for Wolfram syndrome. As I mentioned in my blog on January 1, 2020, I am determined to make 2020 the game-changing year […]
Bringing regenerative therapies to patients
We have been developing drug-based therapies to delay/stop the progression of Wolfram Syndrome, and we have made significant progress. In parallel, we need to develop regenerative therapies to treat/replace damaged tissues. We have two pipelines for the regenerative therapies. 1. Stem cell-based therapies.This requires collaboration with surgeons and bioengineers. We are working on this.2. Regeneration […]
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 […]