Creating More Than Hope
Today is my 40th birthday, and it also marks the fourth anniversary of the Raiden Science Foundation (RSF). It’s a bittersweet milestone for our family. When we launched RSF on a pandemic Thanksgiving in 2021 (November 25), we also shared the “Dear Raiden" video. Looking back at it still breaks my heart, because in those early days, all we had was hope and the rebellious will to fight alongside Raiden for a better future.
Raiden is now five, and life for him hasn’t gotten any easier. There are more medical appointments, more equipment, and more care as his needs grow. While I’m still coping with grief and sadness, I'm also embracing this journey with so much love, hope, and joy. You’ll see that reflected throughout this end-of-year newsletter.
When Raiden was diagnosed with UBA5 disorder, there was barely any research, and little was known about this debilitating rare disease. Back in 2021, there was no clear path, no roadmap, and no hope for a treatment. But through sacrifice, sleepless nights, and refusing to give up, here we are in 2025—with the first treatment for UBA5 disorder now within reach! In just four years, we have blazed a historic trail and redefined what parent-led rare disease drug development looks like. We have been able to move mountains because of the incredible support and generosity of our community.
Together with Dr. Toloo Taghian and the gene therapy research and clinical teams at UMass Chan Medical School, we are creating more than hope. We recently received encouraging feedback from the FDA, and we are now ready to begin the next phase of bringing a promising gene therapy from bench to bedside. To do that, we must raise $2M to manufacture the gene therapy. Without these funds, everything we’ve worked for will be forced to pause❗️
And that is why this moment matters so much. We are closer than ever to a life-changing treatment for UBA5 disorder. As a birthday wish and to celebrate RSF’s fourth anniversary, please consider donating to help us reach this next critical fundraising milestone. If a donation isn’t possible right now, we are always grateful for introductions, ideas, or any form of support. And of course, sharing our story is always deeply appreciated.
Together, let's make the impossible possible
Tommy Pham – Raiden’s Dad
🎁 Donating with crypto or stock? Click here 🎁
Research Updates
Pivotal Milestone for Our Gene Therapy Program
The Pre-Investigational New Drug (Pre-IND) package was submitted to the FDA in October for our groundbreaking UBA5 gene therapy program at UMass Chan Medical School. It was a major step toward developing the first-ever treatment for UBA5.
A Pre-IND is an early part of the regulatory process that allows researchers to share data and plans with the FDA and receive guidance to ensure that safety studies, clinical trial design, and manufacturing plans are on the right path.
This month, we received positive feedback from the FDA and are now ready to begin manufacturing the UBA5 gene therapy as the next phase before clinical trial. But to move forward, we need to raise $2M.
St. Jude's Cutting Edge UBA5 Research
Dr. Heather Mefford and her research team at St. Jude have developed a novel brain organoid model to study UBA5. This research not only deepens the understanding of UBA5 disorder and helps identify treatments, but it also has the potential to advance research for other rare neurological diseases.
Unique Model of rare epileptic disease helps pinpoint potential treatment route
The scientific paper was published in Science Translational Medicine, a leading online journal publishing research at the intersection of science, engineering, and medicine.
Thank you Cure Epilepsy for co-funding the innovative St. Jude UBA5 research in partnership with us. Read more
Ongoing UBA5 Research and Awareness in the Scientific Community
We had a sponsored table at the first-ever MDS-PAS Conference, hosted by Dr. Darius Ebrahimi-Fakhari from Boston Children’s Hospital. The event brought together experts to discuss the latest advancements in pediatric movement disorders.
The drug repurposing team at Oregon Health & Science University, led by Dr. Jonathan Pruneda, has identified three drugs that can restore UBA5 function. Additional studies are now underway.
UBA5 mouse models developed by Baylor College of Medicine continue to undergo viability and behavioral phenotyping (disease symptoms). These models are helping researchers better understand UBA5 disorder. Learn more about the partnership between RSF and the BCM Center for Precision Medicine Models in this NIH article.
Volunteer Spotlight
We are grateful to our two amazing volunteers.
This summer, we welcomed Abby Wen as our Community Engagement Lead. Abby is a student at UC Berkeley, focusing on digital media and performance studies. She led our fundraising efforts at the Jade International Night Market.
Last month, Zaid Amer joined us as our Media Specialist. Zaid is a research technician at Yale, where he works on PROTAC development. He has been creating videos that will help share the stories of UBA5 families in the months ahead.
Upcoming Charity Events
GivingTuesday (12/02/25) – Mark your calendar and consider making a gift to support us. And don’t forget: many employers offer donation matching.
Wreck The Halls (12/13/25) – FightRise is hosting a charity stream to support RSF with many fighting game prizes. 🎮 Link for more details
Shamrock Run (03/15/26) – RSF is an official charity partner of the 2026 Portland Shamrock Run! Follow us for updates on how to join and support.
Community Highlights
Fight4Rare x Mortal Kombat Charity Auction
Mortal Kombat co-creator John Tobias generously donated his time and artwork, and rallied other MK legends and artists to support our auction. 🎥 Recap
Complex featured our event on their IG page on Halloween! Complex is a major digital platform covering entertainment, style, sports, and pop culture. 😮 Check out the story
Spooktacular Fright4Rare Week
Our annual stream week was a success, with the gaming community hosting charity streams and tournaments throughout the last week of October.
💙 Set up a charity stream for us anytime 💙
Big shoutout to ModRetro for donating their Chromatics to support our giveaways and for creating an exclusive RSF edition.
Panda Express Virtual Community Fundraiser
We partnered with Panda Express on October 29 for a nationwide fundraiser. RSF received 50% of all online orders that used the code Raiden. We are so grateful for the support. ❤️🐼 Promo video
A huge thank you to Warner Bros. and Humble Bundle for the incredible summer gaming charity bundles benefiting RSF! 🙌
Featured Charity at Gaming Conventions
We were selected as a charity at DreamHack Dallas in May and featured on the main stage alongside the Rare Village Foundation. 🎥 Recap
It was our fourth year activating at EVO Las Vegas in August. Another big success with amazing support from the fighting game community. 🎥 Recap
Our 4th Annual Run4Raiden
Another successful virtual 5K in March, uniting our community and UBA5 families across the country. This year, we ran for Raiden… for Bryce… for Paisley. 🎥 Recap
Raiden's Adventures
Thank You Make-A-Wish Oregon for Granting Raiden's Wish
It was a truly magical experience. Raiden’s Wish began with an unforgettable Star Wars send-off at PDX Airport on May 4th, followed by a week at the Aulani Disney Resort in Hawaii, where the Phamily made lasting memories and connected with 25 other Wish families during Wish Week. 🎥 Highlights
USA Today – 'Is this real?' How Disney makes wishes come true for critically ill kids
NewsNation – Child with ultrarare genetic disease gets Star Wars day sendoff
The Oregonian – A rebellion of hope: One Portland family fights a rare disease...
Meeting New Friends and Sharing Raiden's Story
During Ryan Trahan and Haley Pham’s 50 States in 50 Days journey (IYKYK), Raiden and the Phamily had the chance to meet them during their stop in Beaveron, Oregon.
Raiden also visited the Tetris headquarters in Honolulu and had the opportunity to meet the wonderful team, including Jaime, Gerilynn, Chris, and their CEO Maya Rogers.
