New York, Jan. 24, 2023 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) Drugs in Development by Stages, Target, MoA, RoA, ...

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 ...

REGENXBIO faces regulatory setbacks for its rare disease drugs, with cash extending only into early 2027. See why RGNX stock ...

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 ...

JCR announced that it will present new clinical data in a poster session at the 18th International Symposium on MPS and Related Lysosomal Diseases.

– Additional Posters Underscore Broad Potential of J-Brain Cargo ® Platform, Including Preclinical Data for Pabinafusp Alfa in MPS II and Nonclinical Data for JR-446 in MPS IIIB – HYOGO, ...

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 ...

Purpose: To evaluate the safety and efficacy of recombinant human iduronate-2-sulfatase (idursulfase) in the treatment of mucopolysaccharidosis II. Methods: Ninety-six mucopolysaccharidosis II ...