A rare and amazing opportunity

As many of you are aware, Science Magazine featured my basic, translational, and clinical research on Wolfram syndrome. The article covers the history of Wolfram syndrome and its research. https://www.sciencemag.org/news/2021/02/race-treat-rare-fatal-syndrome-may-help-others-common-disorders-diabetes https://science.sciencemag.org/content/371/6530/663 This is a rare and amazing opportunity for us to raise awareness of Wolfram syndrome. Science initially interviewed me on endoplasmic reticulum stress (ER […]

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

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

Regenerative Gene Therapy

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

raise awareness

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

patient support group

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

regeneration

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

aris Wolfram syndrome workshop

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

So 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

Patient Care

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

Cure for Wolfram 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. […]

Ready to say goodbye

Hello New Year

Thank you for reading my blog. I always feel your support. I appreciate it immensely. As I am ready to say goodbye to 2017, I would like to update you about our progress. Our drug-repurposing clinical trial of dantrolene sodium is ongoing. The trial began in January, and nineteen patients with Wolfram syndrome from the […]

US Senator Blunt and NCATS Director Dr. Austin visited us from DC

US Senator Roy Blunt (Missouri) and Director of National Center for Advancing Translational Sciences/NIH Dr. Christopher Austin visited us from Washington DC on August 9th. I had a chance to present our Wolfram syndrome clinical and research program to their teams. My colleague in Internal Medicine, Dr. Dan Ory, and I tried to tell them […]

Thank you, Wolfram Research Clinic

Another successful Wolfram research clinic was completed this week. I would like to thank all the participants, Dr. Hershey, Dr. Marshall, Mrs. Samantha Ranck, and other physicians and scientists who were involved in the clinic. See you next year! Thank you again. Warmest regards,Fumi Urano

Clinical trial update: next phase

I have received many questions regarding our ongoing clinical trials, as well as questions related to our next step lately. We started the patient registry, and then moved onto the research clinic. Now, we are conducting trials. I think we are making progress, and need to speed up as we are racing against time. Although we don’t […]

Clinical Trials in the US, UK, and Europe

I have received many emails and phone calls regarding the ongoing clinical trials for Wolfram syndrome in the US, UK, and Europe. After the discussion with Dr. Barrett’s team in the UK and other regulatory experts, we decided to summarize the gist of the ongoing clinical trials. You can find the information on the following […]

Clinical trial update

Dear Friends, I would like to update you about the status of our clinical trial of dantrolene sodium in patients with Wolfram syndrome. Dantrolene is a US Food and Drug Administration (FDA)-approved drug currently used for different medical conditions. Based on our data, we received orphan drug designation of dantrolene sodium for the treatment of […]

Homeostasis

Our current focus is to bring our potential drugs to our patients. We are interested in drugs that can help patients “regain homeostasis.” In other words, we try to help our patients’ organ systems to keep cellular endoplasmic reticulum states stable. The challenge is that there are not many drugs that can modulate endoplasmic reticulum […]

Become a member of the NIH’s Rare Disease Program

We have become one of the first  8 members of the NIH’s Rare Disease Data Repository Program. This is one of the important projects at the National Center for Advancing Translational Sciences (NCATS), National Institutes of Health (NIH) in the USA. I have also become a steering committee member of this program. The purpose of this […]

Thank you for your continued support.

I tried to make progress in the following three areas in 2016: 1) Improve Clinical Care, 2) Raise Awareness, and 3) Develop Novel Treatments for Wolfram syndrome. I did my best. I know I could not make a breakthrough, but I will keep on trying. My highest priority right now is to work out the […]

Wolfram UK Conference and their Clinical Trial plans

UK Wolfram syndrome conference 2016

I attended the Wolfram UK and WellChild conference in England on Saturday, October 8th, 2016. This was my third time at the conference and met with around 100 patients and their family members. At the conference, Dr. Timothy Barrett (University of Birmingham) announced that his phase 2 randomized clinical trial of his repurposed drug in patients […]

Goals and progress in August 2016

I would like to accomplish the following three goals: #1 Improve Clinical Care, #2 Raise Awareness, and #3 Provide a Cure for Wolfram syndrome. I feel that we have been making steady progress in #1 and #2. #3 is the most challenging one. To achieve #3, we need to first delay the progression of the […]

Regenerative medicine symposium

Regenerative Medicine Symposium

I had a chance to present my data and concept on regenerative medicine for the treatment of Wolfram syndrome last week at the regenerative medicine symposium in Tokyo, Japan. Five faculty members from Washington University attended this important symposium. I got constructive feedback from the audience. To achieve a cure, we need to develop regenerative […]

Thank you for the Wolfram Research Clinic

Our annual Wolfram syndrome research clinic took place this week. I would like to thank all the participants, staff members, scientists and doctors who were involved in the clinic. Two clinical trials are planned in the US and UK. I will keep you updated about these. I always appreciate your continued support. Take care,Fumi Urano

Wolfram Syndrome Workshop in France

France 2016

I attended the 6th Wolfram syndrome workshop in Nantes, France last week. I still remember the first workshop which was held in 2009. Since then, we have made significant progress in the past 7 years. Two potential clinical trial plans in the US and UK were discussed. I presented my ideas and plans about a […]

Washington DC, NCATS, and World Orphan Drug Congress

I have been in Washington DC this week to visit the National Center for Advancing Translational Sciences (NCATS) of National Institutes of Health and attend the World Orphan Drug Congress. My trip has been quite fruitful so far. I had a productive discussion with the drug development team at NCATS. We do have new drug […]

Keep on working

I received many emails regarding the status of the drug development. I am sorry that I have not updated you through this site. I am making steady progress. Our current target is “endoplasmic reticulum (ER) calcium homeostasis.” Based on our preclinical studies, we need to maintain high ER calcium levels to delay the progression of […]

Rare Disease Day

Today is a Rare Disease Day. I am thinking of our patients and families suffering from life-threatening diseases in the world. I am very sorry that I have been slow. I will keep on doing my best to develop and bring novel treatments to our patients. I always appreciate your continued supports. I understand that […]

A fruitful trip to Denver

I just came back from my trip to Denver. I attended the fundraising event hosted by the Ellie White Foundation in Boulder on the 17th. I was impressed and moved by Ellie and her mother’s speeches. I could connect with their friends, families, supporters, and other patients. I would like to thank the White family, […]

Thank you, Wolfram syndrome patient organizations in the world

raise awareness

The rare disease day is approaching. I would like express my deep appreciation and gratitude to multiple patient organizations supporting my research. I will keep on working with them to improve clinical care, raise awareness, and develop novel treatment for Wolfram syndrome. Snow Foundation (USA) Ellie White Foundation for Rare Genetic Disorders (USA) Worldwide Society […]

100 lectures on Wolfram in the past 10 years

Last week, I gave a lecture on Wolfram syndrome at the University of Southern California Medical Center. I received constructive comments and feedback. I gave more than 100 lectures on Wolfram syndrome in the past 10 years. I will keep on doing my best to raise awareness of Wolfram syndrome. I was recently asked to […]

Climb the stairs together hand in hand

Wolfram Cure 3 Steps

I would like to begin by thanking all the people around the world who keep on supporting me. I think of you all the time. We climb the stairs together hand in hand to bring novel treatments to our patients. Thank you again. I will not give up. I will keep on doing my best. […]

Thank you, Thank you, Thank you. Our progress.

Wolfram Cure 3 Steps

As we approach the end of this year, I would like to thank you again for your continued support, encouragement, and patience. The number of emails and phone calls I receive has been increasing. Three things are always on my mind.1. Raise Awareness of Wolfram Syndrome2. Improve the Clinical Care3. Provide Novel Treatments and A […]

Snow Foundation’s Wolfram Syndrome Clinical Trials Fund

Snow Foundation’s Wolfram Syndrome Clinical Trials Fund

The Snow Foundation has created the Wolfram Syndrome Clinical Trials Fund. I just looked at videos of our patients and was quite moved. I appreciate their continued support. I cannot thank you enough. We are racing against time.https://snowfunds.org/web/snow/pub I hope you have a wonderful weekend. Take care, Fumi Urano

NeuroRestorative Therapy

Restoring Vision

I am pleased to announce that we have launched the NeuroRestorative Therapy team in the Hope Center, Washington University Medical Center. We are a group of neurosurgeons, neurologists, ophthalmologists, bioengineers, and geneticists like myself. The mission of our team is to restore neurological functions in patients suffering from diseases that impact the human nervous system through […]

Wolfram Syndrome Annual Conference in UK

This year’s Wolfram Syndrome Annual Conference in UK was successful again. I could connect with patients and doctors in UK again. I learned a lot from them. I would like to thank Tracy Lynch, Paul Lynch, Alan Nye, Lottie Nye, Dr. Timothy Barrett, Dr. Patrick Yu Wai Man, Dr. Karen Morrison, and all the patients […]