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Landmark Nature Medicine study reports promising new treatment reduces suffering in Sanfilippo syndrome - EurekAlert
As a neurodegenerative disease characterized by childhood onset dementia, Sanfilippo syndrome causes immense suffering in many ways, including pain, loss of speech, extreme agitation, and distress, gastrointestinal symptoms, and profound sleep disturbance. With no approved treatment, clinical specialists have had few options to help alleviate this suffering until now. A groundbreaking clinical trial collaboration between study lead and principal investigator Lynda Polgreen, MD, MS, Investigator at The Lundquist Institute for Biomedical Innovation at Harbor-UCLA (TLI) and Associate Professor of Pediatrics at the David Geffen School of Medicine at UCLA and Cure Sanfilippo Foundation’s Chief Science Officer and study co-investigator, Cara O’Neill, MD, FAAP, used an innovative approach to treat this disease by targeting neuroinflammation, as it is thought to be a key contributor to disease symptoms.
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