Denali Therapeutics is a promising underdog in the biotech sector, focusing on neurodegenerative diseases, with significant investments in scientific exploration and a robust financial health despite ...

The symptoms are usually seen between 18 and 36 months of age, although there is a wide range of presentation, which is often classified in severe and attenuated forms.

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 ...

JCR Pharmaceuticals Co., Ltd. (TSE 4552; “JCR”), a global specialty biopharmaceutical company dedicated to developing therapies for rare and genetic diseases, announced the launch of a film featured ...

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 ...

Recent successful meeting with the FDA provides path to file for accelerated approval and subsequent conversion to full approval Plan to submit biologics license application (BLA) early in 2025 under ...

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 ...

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 ...

HYOGO, Japan--(BUSINESS WIRE)--JCR Pharmaceuticals Co., Ltd. (TSE 4552; “JCR”), a global specialty biopharmaceutical company dedicated to developing therapies for rare and genetic diseases, today ...