In 2020, we are giving ourselves the means, and accelerating!
Goal: fund a clinical trial
Every step brings us closer to Victory
CACH syndrome affects very young children. An enzyme does not work and prevents cells from making myelin
- motor problems, in particular in walking
- encephalopathy
- irritability
- drowsiness
- seizures
The disrupted cellular mechanisms cause stress in the cells.
Laboratory work has tested drugs capable of reducing cellular stress
A paediatric clinical trial is being prepared at a cost of €500,000, we are on the threshold of the first treatments, ELA’s hope is growing
All together against leukodystrophies
We are counting on you
Because if not you, then who?