Global Roadmap for Sanfilippo Syndrome Therapies
Sanfilippo Children’s Foundation (Australia), Cure Sanfilippo Foundation (USA) and our international sister organisations have collaborated to produce a Global Roadmap for Sanfilippo Syndrome Therapies. In developing this research Roadmap we drew on the expertise of clinicians, researchers, industry leaders, family-led organisations and families from around the globe
The Roadmap has a single purpose - to drive research towards effective therapies and better outcomes for families affected by Sanfilippo syndrome.
The pillars of the Roadmap focus on how to STOP Sanfilippo at its root cause, how to TREAT the underlying impact of the disease on the brain and body, and how to better MANAGE symptoms and improve quality of life. The research themes are underpinned by a set of ENABLING INITIATIVES - data, research tools and collaborative platforms - that could support multiple aspects of research along the entire therapy development pipeline.
Now, with the Roadmap in hand, Sanfilippo patient organisations from around the world will be coming together to focus attention on their shared priorities. We will bring together round-tables of industry and other stakeholders to discuss what data and tools can be shared to accelerate progress for everyone.
Why do we need a roadmap?
In the following video, families and the Sanfilippo Children's Foundation Board talk about the importance of a strategic approach to research in solving Sanfilippo.