Tividenofusp alfa treatment showed reduction and normalization in key disease biomarkers, stabilization or improvement in clinical endpoints ...

The devastating consequences of mucopolysaccharidosis II (MPS II), also called Hunter syndrome, may soon become preventable ...

– Additional Posters Underscore Broad Potential of J-Brain Cargo ® Platform, Including Preclinical Data for Pabinafusp Alfa in MPS II and Nonclinical Data for JR-446 in MPS IIIB – "The data presented ...

Ongoing clinical research at UNC could lead to a first-of-its-kind enzyme replacement therapy for Hunter syndrome, an ultra-rare disorder that causes ...

Additional interim data from the DNL310 Phase 1/2 study will be presented at the SSIEM symposium in August Management will host a webinar for analysts and investors at 8:30 a.m. Eastern Time today ...

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 ...

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 ...

Denali Therapeutics Inc.'s DNL-310 is currently being explored in an ongoing phase 2/3 COMPASS study for treatment of patients with MPS II; Enrollment completion expected in 2024. Denali is in talks ...

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 ...