A coalition of patient advocacy organizations has published a Global Roadmap for Sanfilippo Syndrome Therapies, in an effort to accelerate research and develop effective therapies and care for families affected by Sanfilippo syndrome.
“The Roadmap aims to capture the historical landscape of research and clinical trials, the gaps that we currently see which are limiting progress, and ways in which we can move forward to address them collaboratively,” Cara O’Neill, MD, the chief science officer at Cure Sanfilippo Foundation, co-developer of the strategy plan.
“Notably, we have done this through the lens of impact on the patients and families who are affected,” she said. “We hope the Roadmap creates a level understanding of the evolving state of science regarding Sanfilippo at this time and where work needs to be done to better address those gaps with a patient-centered mindset.”