Epicrispr’s one-time EPI-321 therapy shows early muscle growth after DUX4 silencing
Read how Epicrispr’s early EPI-321 muscle gains could reshape FSHD drug development, and what the small trial still cannot prove.
Pharma & Biotech
Pharma & Biotech
Can Servier’s $2.65bn Edgewise deal reshape the muscular dystrophy drug race?
Servier is buying sevasemten before pivotal muscular dystrophy data. The deal gives Edgewise cash and investors a sharper cardiology story.
Pharma & Biotech
Why BridgeBio Pharma, Inc.’s BBP-418 priority review could redefine the future of LGMD treatment
BridgeBio Pharma, Inc.’s BBP-418 wins FDA Priority Review for LGMD2I/R9. Discover what this could mean for rare muscular dystrophy treatment.
Pharma & Biotech
Why ARTHEx Biotech’s ATX-01 update could reshape RNA-targeted therapy in myotonic dystrophy
Find out how ARTHEx Biotech’s ATX-01 data could reshape RNA-targeted therapy for myotonic dystrophy today!
Pharma & Biotech
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.
Pharma & Biotech
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.
Pharma & Biotech
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.
Pharma & Biotech
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?
Pharma & Biotech
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.
Pharma & Biotech
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.