We Can Save Children's Lives Together

ADSL Deficiency is a complex genetic disorder that causes progressive damage to the brain. In some children, it may look similar to autism or developmental delay. In its most severe forms, it can lead to devastating seizures, loss of developmental skills, and increasing difficulty with basic functions such as eating, moving, and breathing. Families living with ADSL Deficiency face challenges that no child or parent should have to endure.

But it doesn't have to stay this way.

This summer, Rare Birds Foundation is raising $250,000 to close a critical funding gap in a promising gene therapy project at University of Texas Southwestern Medical Center. Without this funding, the project could be delayed or shelved altogether—and our children simply cannot afford to wait.

This gene therapy has the potential to change the future of ADSL Deficiency. For children already living with the disorder, it could halt further progression and preserve the abilities they still have today. For babies diagnosed early enough, treatment may prevent many of the severe seizures, developmental losses, and neurological complications that have defined ADSL Deficiency since it was first identified in 1984.

For the first time, we can envision a future where a diagnosis of ADSL Deficiency is no longer a life-altering tragedy.

Every dollar brings us closer to that future.

If we reach our goal, the program will remain on track to begin treating its first patients within the next few years. Together, we can accelerate a therapy that offers real hope to families around the world—and help ensure that the next generation of children with ADSL Deficiency has a very different story.

Please join us. Together, we can turn hope into a treatment.

Rare Birds Rising

Funding Science that Transforms Lives.

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