JCR Pharmaceuticals Initiates First Dosing in Phase I Clinical Trial of JR-441 for Mucopolysaccharidosis Type IIIA [Yahoo! Finance]
ITOCHU CORP UNSP/ADR (ITOCY)
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Source: Yahoo! Finance
in Japan in the Phase I clinical trial of JR-441, an investigational enzyme replacement therapy for the treatment of mucopolysaccharidosis type IIIA (MPS IIIA, also known as Sanfilippo syndrome type A). JR-441 is a proprietary recombinant heparan N-sulfatase capable of crossing the blood-brain barrier (BBB). MPS IIIA is a rare genetic disorder characterized by severe central nervous system (CNS) symptoms, for which there is currently no approved treatment. Preclinical studies have demonstrated the potential of JR-441 to address CNS-related symptoms associated with MPS IIIA. This open-label, multicenter, single-arm study aims to assess the safety profile, biological effect, and pharmacokinetic profile of JR-441 in patients aged 1 to under 18 years with MPS IIIA. "MPS IIIA has been one of the most significant challenges in the treatment of CNS symptoms," said Dr. Kimitoshi Nakamura, Professor of Pediatrics at the Graduate School of Medical Sciences, Kumamoto University, and the Me
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