We’ve looked at the global research landscape for Sanfilippo, interviewed experts, consulted with families and created a plan to solve Sanfilippo. Behind this plan is an enormous amount of information and research expertise.
The plan includes funding research projects aimed at stopping the progression of the disease, treating the impact on the brain and body, and managing quality of life. We’ll also invest in vital research infrastructure that will enable the science to progress as urgently as possible.
Why do we need a roadmap?
In the following video, families and our Board talk about the importance of a strategic approach to research in solving Sanfilippo.