A treatment for children with SPG4 is closer than ever.
The science is moving. The right partners are in place. What happens next depends on what we do now.
We are Building the Path to Treatment
For the first time, we are in a position to actively move therapies for children with SPG4 forward. With the right funding, this work moves from models and data to treatments that can be tested, refined, and ultimately delivered to children.
-
Building the Models
We are developing the preclinical models needed to test potential therapies and understand how SPG4 progresses.
-
Measuring the Disease
We are advancing biomarker and natural history work to track progression and measure whether treatments are working.
-
Advancing Treatments
We are pursuing multiple therapeutic approaches, including gene therapy and gene editing, to identify the most effective path forward.
The Lilly and Blair Foundation is dedicated to improving the lives of children with de novo SPG4 by bringing together the right people, funding the right work, and accelerating progress toward real treatments. This work is active, coordinated, and already underway. But it does not happen on its own. It happens because people choose to make it possible.
Your support accelerates how quickly this progress leads to real treatments. What happens next depends on how quickly this work is funded.
From Our Story to Theirs
Introduction to The Lilly and Blair Foundation
Washington Post Live Rare Disease Day Interview
