Help make an LBSL gene therapy reality!

This February, in honor of Rare Disease Day on Feb. 28, help us accelerate hope by supporting a new LBSL research study that will launch this year! All funds raised this month will go toward funding MRS scans at study sites that will look at the brain and spinal cord of LBSL patients to determine whether lactate is a LBSL biomarker and support the selection of clinical trial endpoints, as well as funding grants for patients and caregivers to travel to the study sites.

ABOUT ACCELERATE HOPE & CURE LBSL

What started as A Cure for Ellie in 2013 has turned into a global movement on the verge of a groundbreaking, life-changing therapy for patients. Our Accelerate Hope initiative aims to raise $3 million to bring a gene therapy for LBSL into clinical trials.

This therapy is our community’s greatest hope to stop disease progression and change the future for children and adults with LBSL. We've seen encouraging outcomes in the lab. Now, we need the resources to finish the journey. For families living with this devastating leukodystrophy, time is myelin — every second that ticks by is irreversible disease progression — and we cannot afford to wait.

Why give now?

Every donation will be doubled up to $1,500,000 — and your support directly accelerates the path from the lab to the clinic. Every dollar brings us closer to the first-ever treatment option for LBSL.

How gifts to Accelerate Hope will be used:

• Funding toxicology and safety studies required by the FDA before a clinical trial on our gene therapy
• Manufacturing of clinical-grade gene therapy for trial use
• Supporting studies to establish biomarkers and outcomes that will be measured during the trial
• Regulatory preparation and trial readiness to open the first human study

With your help, we can turn hope into action — and action into a cure. Accelerate hope with us!