Zacks Investment Research on MSN

Regenxbio shares rise on plans to resubmit Navsunli BLA for MPS II

Regenxbio RGNX announced that it has reached alignment with the FDA on the next regulatory steps for the potential ...
Nasdaq

FDA Accepts Biologics License Application for Mucopolysaccharidosis II Treatment

PARAMUS, N.J., May 14, 2025 /PRNewswire/ -- 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 ...
Yahoo Finance

Denali Therapeutics Announces FDA Review Extension of BLA for Tividenofusp Alfa for the Treatment of MPS II (Hunter Syndrome)

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The New England Journal of Medicine

An Intravenous Brain-Penetrant Enzyme Therapy for Mucopolysaccharidosis II

Tividenofusp alfa, comprising iduronate-2-sulfatase fused to an engineered transferrin receptor–binding Fc domain, has been developed to treat neurologic and peripheral manifestations of ...
GEN

Sangamo Genome Editing Trial, Assessing MPS II Candidate, Treats First Patient

Sangamo Therapeutics said today it has treated the first patient in the Phase I/II CHAMPIONS clinical trial assessing its in vivo genome-editing candidate therapy SB-913 for mucopolysaccharidosis type ...
BBC

'We don't know how a rare illness will affect our boy'

Three-year-old Wilfred is taken to London weekly to receive treatment for his MPS II disease A year ago, the family of three-year-old Wilfred had never heard of Mucopolysaccharide (MPS) - but now they ...
PharmiWeb

Actigen Initiates a Development Programme in Rare Disease MPS II

Biotech company Actigen initiates a clinical development programme for GNR-055, a potentially breakthrough treatment for the life-limiting, rare disease mucopolysaccharidosis II (MPS II) (also known ...