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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 accelerated approval of Navsunli (clemidsogene lanparvovec-sngl, RGX-121), a one-time ...
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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 ...
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 ...
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 ...
Over 49 weeks of DNL310 (ETV:IDS) treatment in the Phase 1/2 study, positive changes across measures of exploratory clinical outcomes including VABS-II (adaptive behavior) and BSID-III (cognitive ...
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