Rare disease.
Real hope.
Cure LBSL funds research and supports patients and families around the world — and we’re closer than ever to a gene therapy.
What is LBSL?
LBSL stands for Leukoencephalopathy with Brainstem and Spinal Cord Involvement and Lactate Elevation. It’s a rare genetic disorder that affects the brain and spinal cord. There are thought to be 500 patients identified worldwide, but many people go undiagnosed or misdiagnosed.
A Cure in Progress
Since our foundation’s inception in 2013, we’ve been racing to find a cure for LBSL. The research we’ve funded has led us to our main effort: a DARS2 AAV9 gene therapy, led by our partners at Kennedy Krieger Institute in Baltimore.