What PepGen’s 5 mg/kg FREEDOM2 cohort reveals about the risk-reward profile of PGN-EDODM1
PepGen’s FREEDOM2 data hint at PGN-EDODM1 activity in DM1, but bigger questions remain. Read what the 5 mg/kg cohort really changes.
Can Sarepta Therapeutics prove RNA interference works in FSHD1 and DM1 with early Phase 1/2 data
Read why Sarepta Therapeutics’ first siRNA data in FSHD1 and DM1 could matter for rare disease drug development and investor sentiment.
What Avidity Biosciences Phase 1/2 MARINA results reveal about the future of DM1 drug development
NEJM publication of Avidity Biosciences delpacibart etedesiran data raises new questions for DM1 treatment. Discover what this changes next.