Rugonersen, a potentially best-in-class antisense oligonucleotide (ASO) therapy in development for Angelman syndrome, advanced into the pivotal Phase 3 BEACON clinical trial
Angelman syndrome affects approximately 30,000 diagnosed individuals in the US and EU5 and has no approved treatments
Topline data expected in early 2029
July 28, 2026 -- OHB Pediatrics Ltd. d/b/a Oak Hill Bio, a clinical-stage rare disease therapeutics company, today announced that the first participant has been dosed in B