Insights from Wedbush analysts about the clinical effectiveness of REGENXBIO's RGX-202, market potential, and challenges posed by competitors like Sarepta Therapeutics and Pfizer.
New data show additional improvement and stabilization in clinical outcomes in MPS II with 104 weeks of tividenofusp alfa (DNL310) treatment in the open-label, single-arm Phase 1/2 study; sustained normalization of CSF