We would like to share an important update on our ongoing mouse study investigating Venglustat as a possible treatment for the HSP subtypes SPG11, SPG15 and SPG48. SPG11, SPG15 en SPG48.
Earlier studies showed that Venglustat can reduce the build-up of harmful fats in nerve cells and improve movement and behavior in affected mice. In our current study, we are testing different doses and treatment durations to find out what is safe and effective in the long term.
What do the first results show?
The interim results suggest that a very high dose is less effective over longer periods than expected. This is an important and valuable finding. As a result, we have adjusted the study design and added an extra group of mice receiving a lower dose for a longer time. This will help us find the best balance between effect and safety.
Why is this important?
Finding the right dose is a key step in drug development. The knowledge we gain now is essential to safely translate this treatment from the laboratory to patients.
Support needed
So far, Euro-HSP and Life4HSP have raised around €200,000, but another €100,000 is needed to complete the study. Your support is crucial to continue this promising research.
Read here the full study summary in Dutch en English translation here
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