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 ...
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 last time I spoke about REGENXBIO Inc. (NASDAQ:RGNX) it was in a Seeking Alpha article entitled "Regenxbio: RGX-121 Could Achieve A First For Rare Neurodegenerative Disorder". Regarding this ...
Denali Therapeutics (($DNLI)) announced an update on their ongoing clinical study. Denali Therapeutics has launched an open-label extension study ...
FDA will consider an accelerated approval pathway for RGX-121; BLA filing expected in 2024 Pivotal program is active and enrolling patients RGX-121 is a potential first-in-class, one-time gene therapy ...
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