RAIDEN SCIENCE FOUNDATION
MORE THAN HOPE FOR KIDS
WITH UBA5 TO HAVE A
We are overcoming research barriers in order to advance treatment for UBA5, a debilitating and ultra-rare genetic disorder.
In doing so, we are pushing forward medical breakthroughs like gene therapy with the goal of helping millions of kids all around the world who are suffering from rare diseases.
WHAT IS UBA5?
UBA5 IS A LIFE-THREATENING PROGRESSIVE NEUROLOGICAL DISORDER
The Ubiquitin-like modifier-activity enzyme 5 (UBA5) gene contains instructions for the UBA5 protein, which is critical for cellular breakdown and function.
Mutations in the UBA5 gene can result in protein malfunction and even disease.
Diseases associated UBA5 include Spinocerebellar Ataxia and Early-Onset Epileptic Encephalopathy.
FACTS & FINDINGS
There are 30 diagnosed cases in the world, making UBA5 an ultra-rare and subsequently under-researched area of medicine.
Motor control issues include: Hypotonia (floppiness), Spasticity (stiffness), and movement disorder.
Cognitive issues include : seizures, intellectual disability, and brain abnormalities.
Growth issues include: microcephaly (poor head growth) and failure to thrive.
UBA5 HITS CLOSE TO HOME FOR US
Watch our story and learn about what inspires us to help others like us.
OUR RESEARCH + PROCESS
We are taking a holistic approach towards developing effective treatments for UBA5 with a focus on gene therapy as our first solution. We will be operating like a lean startup, meaning we will be fast, efficient and cross-functional. We know that our work will ultimately benefit countless kids suffering from numerous rare diseases around the world.
DELIVER, DEVELOP, DELIVER
WE’RE GRATEFUL FOR ALL
THE SUPPORT ON OUR JOURNEY 🙏
MEET DR. AARON CIECHANOVER
2004 Nobel Prize award winner in Chemistry for the discovery of ubiquitin pathway (of which UBA5 is a part of) and a member of our Scientific Advisory Committee.
"The discovery of the ubiquitin system was driven by sheer curiosity – how the cell degrades its own damaged proteins and those [proteins] that have completed their role.
But as happens frequently, curiosity led to the discovery of numerous diseases caused by aberrations in the [ubiquitin] system, and subsequently to discovery of drugs to combat some of them including cancer. It is now our role to complete the cycle and discover remedies to the still orphan diseases – UBA5 mutations among them."
MEET U.S. SENATOR RON WYDEN
U.S. Senate from Oregon and Chairman of the Senate Finance Committee and a senior member of the Energy and Natural Resources Committee.
"Tommy's son Raiden was diagnosed with UBA5, a super-rare genetic disorder. Very few can empathize with the difficulty of parenting a child with this genetic disorder, but Tommy has
mobilized this moment as an opportunity to help others." Senator Ron Wyden said. "The Raiden Science Foundation epitomizes the Oregon spirit of tenacity through crisis, and has the potential to lift up the community in the process.
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