Promising Mucopolysaccharidosis Type I Pipeline Therapies such as Laronidase, Antihistamine, Antipyretic, JR-171, AGT-181, ...

Mucopolysaccharidosis Type I Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage ...

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 ...

Why Tennessee softball signee and Gordonsville pitcher Kailey Plumlee kept playing, how she coped with knee surgery last ...

BioMarin has been resetting its business with cuts in headcount and a narrowing of active programmes over the last year or so, but is now looking to the future with a takeover deal for Inozyme Pharma ...

Simpson, NASDAQ: RGNX) for RGX-121 (clemidsogene lanparvovec), a potential first-in-class, investigational gene therapy for the treatment of Mucopolysaccharidosis II (MPS II). The FDA granted ...

Title: Autologous human peripheral blood B cells genetically engineered to express human iduronidase: Results from a first-in-human clinical trial in subjects with mucopolysaccharidosis type I ...

In May 2025, Denali Therapeutics Inc. announced a Phase 2/3 study of the efficacy and safety of tividenofusp alfa (DNL310), ...

To help raise awareness about MPS, Kingston City Hall and Springer Market Square will be lit up in blue and yellow on May 15.