He has Ultra-Rare UBA5 Disease, but There is Hope for this Little Boy


Little 2 1/2-year-old Raiden Pham cannot walk or talk, but he can smile at his parents and radiate love. The baby’s parents are on a 24/7 schedule caring for him. Raiden, who must be tube-fed, cannot sit independently or control his head movement.

Yet his devoted parents, while managing full-time jobs, are determined to fulfill his every need and find a cure for his rare disease. Linda, Raiden’s mother, describes the day they found out that there was no cure. She acknowledged that it was the darkest day of their lives. Their hearts were broken.

KPTV Portland recently carried a short story about Raiden, his devoted parents Tommy and Linda, and his older sister Jordan.

At first glance, the viewer may not even notice that Raiden has UBA5, a rare genetic disease.

After years of testing, Tommy and Linda received a diagnosis in 2021. Linda offered a list of the tests administered while searching for the cause of Raiden’s disorder:

  • Chromosomal Array
  • Mitochondrial DNA-NGS Panel
  • Biotinidase and plasma amino acid
  • NextGen for long-chain fatty acid disorders
  • Comprehensive Intellectual Disability Panel
  • Skin biopsy for fibroblast to investigate ACAD10
  • Whole Exome Sequencing Test (one of the most in-depth tests available)

The results were not decisive. There was a suggestion that Raiden may have a mutation outside of coding regions or in a non-coding region of RNA transcript that may not be identified by whole exome sequencing.

The parents have created an organization called the Raiden Science Foundation to raise money and awareness for this extremely rare and life-threatening disease.

Recently the Foundation signed off on an $85,000 gift agreement with the University of Massachusetts Chan Medical School to jump-start the research.

Linda admits that even with their work and devotion, a cure may not be found, or his life may be shortened by the illness. These thoughts haunt her night and day.

Where There’s Life . . .

Ruth Napier, M.D., an Assistant Professor at Oregon Health & Science University, offers hope through the funding of the first gene therapy for UBA5.

The Paradigm

Dr. Napier explained that:

  • We have two copies of most genes
  • genes instruct the body to create proteins
  • UBA5 gives instructions to make an enzyme involved in attaching ubiquitin to proteins.
  • Ubiquitin, a compound in living cells, is responsible for the destruction of defective proteins.

 But if both copies of the gene have mutations (changes) that would interfere with the gene’s functioning and the body would become deficient.

Dr. Napier explained that treatment will be administered to children giving them a non-mutated type of enzyme in the central nervous system.

Dr. Napier said that ultimately, they hope to restore the children’s health. There is always hope.

Raiden’s father added that he feels this is not just hope but it can become a reality.

The Trailblazers Join the Fight

The Phams will be headlined at a Sunday Trailblazer game at halftime. Fans can donate or pledge donations according to the number of three-pointers made by either team. Pledges can be made to the Raiden Science Foundation.



Rose Duesterwald

Rose Duesterwald

Rose became acquainted with Patient Worthy after her husband was diagnosed with Acute Myeloid Leukemia (AML) six years ago. During this period of partial remission, Rose researched investigational drugs to be prepared in the event of a relapse. Her husband died February 12, 2021 with a rare and unexplained occurrence of liver cancer possibly unrelated to AML.

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