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.
Sarepta’s sirolimus gambit: Can enhanced immunosuppression rescue ELEVIDYS for non-ambulatory Duchenne patients?
Sarepta’s ELEVIDYS Cohort 8 targets non-ambulatory Duchenne patients with a sirolimus regimen. What the trial design reveals about the path to label restoration.
BridgeBio Pharma advances BBP-418 toward FDA filing after Phase 3 muscular dystrophy trial data
BridgeBio Pharma reports Phase 3 BBP-418 results and prepares FDA filing. Could this become the first therapy for limb-girdle muscular dystrophy?
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.
Scribe Therapeutics deepens in vivo CRISPR validation with second Eli Lilly collaboration milestone
Scribe Therapeutics hits a second in vivo CRISPR milestone with Eli Lilly. Explore what this changes for gene editing platforms and future therapies.
Can Sarepta’s ELEVIDYS redefine Duchenne therapy? Inside the 3-year EMBARK data
Sarepta’s gene therapy ELEVIDYS shows 3-year durability in Duchenne muscular dystrophy. Find out what it means for patients, payers, and global rollout.
What does Swissmedic approval of Santhera’s AGAMREE mean for long-term steroid treatment in Duchenne muscular dystrophy?
Swissmedic approves Santhera’s AGAMREE for Duchenne muscular dystrophy. Find out how vamorolone could reshape long-term steroid treatment strategies.