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Advocacy Groups Unveil ‘Roadmap’ for Sanfilippo Research, Care
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, said in a press release. O’Neill, the chief science officer at Cure Sanfilippo Foundation, is a co-developer of the strategy plan.
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