Antisense oligonucleotides reverse rare neurodevelopmental disorder in preclinical models
Scientists at St. Jude Children’s Research Hospital have found they can reverse the effects of HNRNPH2-related neurodevelopmental disorder using…
PepGen Announces Topline Results from Lowest Dose (5 mg/kg) MAD Cohort in the Ongoing Phase 2 FREEDOM2 Study Demonstrating Favorable Safety, Splicing and vHOT Data
– PGN-EDODM1 was generally well-tolerated with all adverse events mild or moderate and no serious adverse events reported…