12-month pivotal data further demonstrate the ability of one-time RGX-121 treatment to improve outcomes for patients with MPS II >80% reduction in CSF levels of HS D2S6, key biomarker of MPS II brain ...
Denali Therapeutics Inc. (DNLI) is advancing a drug by the name of DNL-310 [tividenofusp alfa] for the treatment of patients with MSP II [Hunter Syndrome]. It had been able to report positive results ...
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 ...
FDA issues Complete Response Letter for RGX-121 (clemidsogene lanparvovec) for treatment of Mucopolysaccharidosis II (MPS II) REGENXBIO plans to work with FDA on a path forward with the goal of ...
ROCKVILLE, Md., Feb. 8, 2021 /PRNewswire/ — REGENXBIO Inc. (Nasdaq: RGNX) today announced that additional positive interim data from a total of eight patients in Cohorts 1 and 2 of the ongoing Phase I ...
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 ...
The Food and Drug Administration (FDA) has accepted for Priority Review the Biologics License Application (BLA) for tividenofusp alfa for the treatment of Hunter syndrome, also known as ...
Analysis from continued follow-up of Phase 1/2 clinical trial data in Hunter syndrome (MPS II) reinforces potential for tividenofusp alfa (DNL310) to address full disease spectrum Launch readiness ...
The FDA clinical hold follows a report of a single central nervous system neoplasm in a participant treated with RGX-111 four years prior. RegenxBio’s investigational gene therapies, RGX-111 and ...
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