New Research Will Test CLR01 ‘Molecular Tweezer’ in Sanfilippo Models

New Research Will Test CLR01 ‘Molecular Tweezer’ in Sanfilippo Models

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New Research Will Test CLR01 ‘Molecular Tweezer’ in Sanfilippo Models

A newly funded research study will test whether an investigational therapy dubbed a “molecular tweezer” — officially called CLR01 — might ease symptoms of Sanfilippo syndrome in preclinical models. The project is co-funded by the Cure Sanfilippo Foundation, the Australian Sanfilippo Children’s Foundation, and the HANDS consortium of patient groups, scientists, and clinicians. “The hope is that with promising results, we can move to a clinical trial for treating children with CLR01,” Cara O’Neill, MD,…

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