Gene Therapy Offers Hope for Bubble Boy Disease

Quick Smiles:

• Groundbreaking gene therapy remains 95% effective in treating ADA-SCID, or “Bubble Boy disease.”
• Children like Eliana Nachem have enjoyed healthy, active lives after treatment.
• FDA approval is on the horizon for this life-changing therapy thanks to strong long-term results.

Long-term follow-up shows that gene therapy for severe combined immunodeficiency (ADA-SCID) continues to help the majority of treated children lead healthy, normal lives.

In a study spanning several years, 95% of babies and toddlers treated with the therapy maintained fully reconstructed immune systems, allowing them to grow up safely.

“The durability of immune function, the consistency over time and the continued safety profile are all incredibly encouraging,”

said Dr. Donald Kohn, senior author and pediatric transplant physician at UCLA.

The therapy involves correcting the defective ADA gene in a child’s stem cells, enabling the immune system to fight infections that were previously life-threatening.

Between all trials, every patient is alive and thriving, with most showing permanent correction of their immune system.

“Our goal is to have this therapy FDA-approved within two to three years,”

Kohn added. “The clinical data strongly supports approval—now we need to demonstrate that we can manufacture the treatment under commercial pharmaceutical standards.”

One inspiring example is 11-year-old Eliana Nachem, who now enjoys school, dreams of becoming an artist, and lives a vibrant childhood after years in isolation due to her condition.

“I am eternally grateful to every single scientist, doctor, lab worker, nurse, hospital security guard—all the people who had anything to do with this gene therapy coming into existence and saving her,”

her mother, Caroline, shared joyfully.

With unwavering dedication from researchers, children with ADA-SCID now have promising, ordinary futures—proof that hope and determination can transform lives.

Stay tuned for more inspiring breakthroughs that are rewriting the story for families everywhere!

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