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RAIDEN SCIENCE FOUNDATION

Creating more than hope
giving kids suffering from UBA5 Disorder a fighting chance.

We are overcoming traditional research barriers to advance treatment for UBA5 Disorder, a debilitating and ultra-rare genetic disease.

In doing so, we are pushing forward medical breakthroughs, such as gene therapy, to help children all around the world who are suffering from UBA5 Disorder and, ultimately, other rare diseases.

WHAT IS UBA5?
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UBA5 Disorder is a neurodegenerative condition

UBA5 itself is a gene that encodes instructions critical for cellular breakdown and function. Mutations in the UBA5 gene result in protein malfunction, posing life-threatening risks and a reduction in quality of life as early as infancy.

An ultra-rare genetic disease

With only about 40 diagnosed children worldwide, UBA5 Disorder is considered an ultra-rare condition, leading to limited research within the field of medicine. Nevertheless, we are changing that.

THE SYMPTOMS
Motor Control Issues
  • Hypotonia (body floppiness)

  • Spasticity (body stiffness)

  • Dystonia (involuntary muscle movement)

  • Ataxia (no body coordination)

Cognitive Complications
  • Seizures (epilepsy)

  • Global developmental delay 

  • Intellectual disability

  • Other brain abnormalities

Other Medical Challenges
  • Microcephaly (poor head growth)
  • Gastrointestinal (fed via a tube)
  • Severe reflux
  • Respiratory
  • Vision impairment
  • Requires 24/7 care 
     
Every UBA5 child is unique, and the symptoms manifest differently over time

UBA5 hits close to home for us

Watch our hopeful story and learn about what inspires us to create a better tomorrow

RARE IS NOT SO RARE

200M

Children are affected by rare disease worldwide 

60M

children affected by a rare disease will not live past 5 years old

95%

of rare diseases have no approved treatments

80%

of rare diseases are caused by genetics defects

The research we are accelerating today will have a scalable impact across other rare diseases tomorrow

So what can you do to help?

Latest Updates
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Space4Rare

As part of the historic lunar landing on February 22nd, 2024, our journey and Raiden's story are now officially preserved on the moon for years to come, inspiring hope and possibilities.

Read more

Join our Fight4Rare community and support the fight against rare diseases.

Wear Your Support

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We're grateful for all
the support on our journey.

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