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Pharmabiz3d
US FDA accepts and grants priority review status to Regenxbio’s BLA for clemidsogene lanparvovec to treat Mucopolysaccharidosis II
US FDA accepts and grants priority review status to Regenxbio’s BLA for clemidsogene lanparvovec to treat Mucopolysaccharidosis II: Rockville, Maryland Thursday, May 15, 2025, 1 ...
FDA Accepts Biologics License Application for Mucopolysaccharidosis II Treatment
NS Pharma, Inc. (NS Pharma), a subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku) announced today that the U.S. Food and Drug Administration has accepted for review the Biologics License ...
Monthly Prescribing Reference5d
Gene Therapy for Hunter Syndrome Under FDA Review
MPS II is a rare, X-linked recessive disorder caused by lysosomal iduronate-2-sulfatase (I2S) enzyme deficiency, leading to glycosaminoglycan accumulation and organ dysfunction. RGX-121 is a one ...
REGENXBIO Announces FDA Acceptance and Priority Review of the BLA for RGX-121 for MPS II
REGENXBIO Inc. (Nasdaq: RGNX) today announced the U.S. Food and Drug Administration (FDA) has accepted for review the Biologics License Application (BLA) seeking accelerated approval for clemidsogene ...
NS Pharma, Inc.: FDA Accepts Biologics License Application for Mucopolysaccharidosis II Treatment
NS Pharma, Inc. (NS Pharma), a subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku) announced today that the U.S.
Investing5d
FDA sets review date for potential Hunter syndrome gene therapy
RGX-121 is designed to deliver the iduronate-2-sulfatase (IDS) gene directly to the central nervous system, potentially offering a permanent solution for the production of the I2S enzyme that ...
Investing5d
FDA sets review date for potential Hunter syndrome gene therapy
RGX-121 is designed to deliver the iduronate-2-sulfatase (IDS) gene directly to the central nervous system, potentially offering a permanent solution for the production of the I2S enzyme that patients ...