Analysis from continued follow-up of Phase 1/2 clinical trial data in Hunter syndrome (MPS II) reinforces potential for tividenofusp alfa ...

Investor enthusiasm and evolving FDA pathways are accelerating rare disease drug development, with ultrarare conditions like ...

Studies of Hunter and Hurler syndromes on hold ...

JCR Pharmaceuticals Co., Ltd. (TSE 4552; "JCR"), a global specialty biopharmaceutical company dedicated to developing therapies for rare and genetic diseases, today announced the presentation of four ...

REGENXBIO Inc. (Nasdaq: RGNX) today announced that the U.S. Food and Drug Administration (FDA) placed a clinical hold on its ...

An AAV vector genome integration event associated with PLAG1 overexpression was identified in a preliminary analysis of the resected tumor.

Hunter syndrome (MPS II) presentations will include analysis from continued follow-up of Phase 1/2 data for tividenofusp alfa (DNL310), currently ...

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 ...

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 ...

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 ...