BEAVERTON, OR (KOIN) – Just 21 months ago, Tommy and Linda Pham welcomed their son Raiden into this world. But when he was 3 months old, they noticed something was wrong.
“He wasn’t hitting milestones. He started puking a lot. I thought it was me. I had so much milk, so the doctor said [to] try the bottle and I did that, but he was still puking 10 times a day,” Linda told KOIN.
What followed for the Phams was a series of medical visits and tests, but doctors weren’t able to pinpoint what was going on until this past summer. That’s when the Phams had all 20,000 of Raiden’s genes tested.
This time, the doctors found something.
Raiden has a UBA-5 gene mutation, a life-threatening progressive neurological disorder.
“There’s not a lot known about it. It’s ultra rare. It’s beyond rare,” Linda said. “Only 30 or 40 cases in the world.”
There’s no treatment. And no cure.
After a month of being in a dark place, Tommy said he started using his contacts in the medical research field and eventually heard from the University of Massachusetts. Their researchers, he said, were interested in coming up with gene replacement therapy for UBA-5.
The Phams jumped on the opportunity and formed the Raiden Science Foundation to raise money for the research.
“We decided to launch on Thanksgiving,” Tommy said. “Oh my God, the amount of support, just a small community. People are re-sharing, re-posting.”
They need $1 million to get going. As of Friday, just weeks into the effort, they had raised more than $165,000.
Officials with U-Mass told KOIN the money raised would be likely used for three purposes: funding research to develop gene therapy vectors specific to UBA5; hiring scientific research staff who would be dedicated to this project; and conducting initial, pre-clinical studies for efficacy and safety.
Their efforts are boosting Tommy and Linda’s spirits, even with the uncertain future for Raiden.
“As a mom it’s, like, there’s nothing you can do but just hope,” Linda said.
Over time, the Phams will likely need to raise $2–$3 million. With ultra rare cases, it’s common for families to get involved in fundraising, since there is typically no government funding.