JCR Pharmaceuticals Announces Completion of Clinical Trial Notification Process in Japan for Phase I Study of JR-441 for Mucopolysaccharidosis Type IIIA [Yahoo! Finance]
ITOCHU CORP UNSP/ADR (ITOCY)
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itochu.co.jp/en/ir/index.html
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Source: Yahoo! Finance
the completion of the regulatory review by the Pharmaceuticals and Medical Devices Agency (PMDA) for the clinical trial notification for the Phase I study of JR-441 in individuals with mucopolysaccharidosis type IIIA (MPS IIIA; Sanfilippo syndrome type A). JR-441 is a recombinant form of heparan N-sulfatase that is developed using JCR's proprietary J-Brain Cargo ® blood-brain barrier (BBB)-penetrating technology. JR-441 addresses a significant unmet medical need by crossing the BBB and targeting the neurological symptoms of MPS IIIA within the central nervous system. "MPS IIIA is a rare and life-threatening lysosomal storage disorder for which there are currently no approved treatment options for individuals living with this devastating disease, and there is a dire need to address the neurological signs and symptoms of MPS IIIA," said Shin Ashida, Chairman and President of JCR Pharmaceuticals. "We are pleased that the PMDA's review of the Phase I study plan is complete, and that c
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