Rare Disease Day at NIH 2024

I’m excited to share some fantastic news with you. I’ve received an invitation to present our research on Wolfram Syndrome at the Rare Disease Day event held at the National Institutes of Health on February 29, 2024. This event is widely regarded as one of the most prestigious gatherings for rare diseases, offering an excellent […]

Rare Disease Podcast

I had an interview for the Rare Disease Podcast in December. You can listen or watch it by clicking on the link below: Rare Disease Podcast Interview

Wolfram Syndrome Documentary – PBS

The Wolfram syndrome documentary, which features Ms. Ellie White, was broadcast on PBS last year. It is now available for viewing on YouTube. I feel privileged to have been involved in this documentary, and I encourage you to watch it and share the link with your friends to help raise awareness about Wolfram syndrome.

Our achievements in Wolfram syndrome research over the past year, in 2023

Our achievements in Wolfram syndrome research over the past year, in 2023
Dear Friends, I hope you enjoyed a wonderful holiday season. I want to take a moment to express my deep gratitude for your unwavering belief in and support of our mission to find a cure for Wolfram syndrome. Your enduring encouragement has been a beacon of hope guiding us on this remarkable journey. As we […]

Wolfram Syndrome UK Conference 2023

Wolfram Syndrome UK Conference 2023
I am thrilled to share my experience attending the Wolfram Syndrome UK conference 2023, which was held on September 30, 2023, to honor the Global Wolfram Syndrome Day on October 1st. It was a wonderful opportunity to present our therapeutic developments and ongoing and future clinical trial plans for Wolfram syndrome, and I was delighted […]

Therapeutic development – Progress Report

September 7, 2023 Dear Friends, Thank you so much for continually believing in and supporting me and our therapeutic development for Wolfram syndrome. I deeply appreciate it. Your encouragement has been such a driving force in our journey. Excitingly, we’re making significant strides in our therapeutic development for Wolfram syndrome. Ongoing clinical trial In collaboration […]

The Power of Hope: Lessons from Patients with Wolfram Syndrome

The Power of Hope: Lessons from Patients with Wolfram Syndrome
Recently, I saw two patients with Wolfram syndrome from Central America. Despite facing a debilitating disease, they had an inspiring positive attitude and gave me two wooden glasses, expressing their faith in me to find an effective treatment soon. This experience reminded me of the importance of hope and optimism in healthcare and how our […]

Highlights from the 8th International Wolfram Syndrome Symposium: Hope on the Horizon

Highlights from the 8th International Wolfram Syndrome Symposium: Hope on the Horizon
The 8th International Wolfram Syndrome Symposium in London was a significant event for researchers and clinicians focused on this rare genetic disorder. The symposium fostered new collaborations and showcased the latest findings in Wolfram Syndrome research. We express our sincere appreciation to Stephanie Snow Gebel and Saad Naseer, MD, from the Snow Foundation, as well […]

United for a Cure: The International Wolfram Syndrome Symposium in London

United for a Cure: The International Wolfram Syndrome Symposium in London
The International Wolfram Syndrome Symposium begins tonight in London, bringing together 25 experts, including physicians, scientists, industry partners, and patient advocacy representatives, to explore cutting-edge treatments and strategies for this rare disease. Discussions will focus on enhancing patient outcomes, engaging the medical community, and advocating for public policy changes. Stay tuned, as I’ll be providing […]

A Ray of Hope: a girl of Israel

At our WFS1 clinic, we have the privilege of meeting extraordinary individuals who inspire and motivate us to push the boundaries of our Wolfram research. Today, I would like to share the story of a remarkable young girl from Israel who visited our clinic, leaving an indelible mark on our hearts and minds. Her courage, […]

First dose

We are excited to report that the Phase 2 Clinical Trial of AMX0035 for the Treatment of Wolfram Syndrome has commenced, with the first participant already dosed earlier this week. This marks our second clinical trial for Wolfram Syndrome, and we are incredibly grateful for the unwavering support from our patients, their families, and the […]

The Heartwarming Spirit of a Young Soccer Fan: A Glimpse into the Life of a 10-Year-Old with WFS1-Related Disorder

Last week, our clinical research unit welcomed a 10-year-old boy with WFS1-related disorder and his parents, who kindly donated their biological samples to support our research. The boy’s passion for soccer and life touched our hearts, and we want to share this heartwarming experience. The boy arrived wearing a Manchester City jersey, sparking conversation about […]