RegenXBio shared positive topline results from their pivotal Phase 3 AFFINITY DUCHENNE study of RGX-202 in 31 individuals with Duchenne aged 1 year and older. The study met the primary […]
Entrada Therapeutics, who received early funding from CureDuchenne to advance their Endosomal Escape Vehicle Platform, today announced positive initial data from their Phase 1/2 ELEVATE-44-201 study in DMD patients amenable […]
What Sarepta Therapeutics’ sNDA submission means for Duchenne Sarepta Therapeutics is preparing to submit supplemental new drug applications (sNDAs) to the FDA for converting the accelerated approvals of AMONDYS 45 […]
Sarepta therapeutics is seeking to enroll 25 non-ambulant individuals with Duchenne in a clinical trial evaluating the use of sirolimus as part of an enhanced immunosuppressive regimen prior to and […]
As an early funder of Capricor Therapeutics, CureDuchenne is pleased to share that the FDA has set the PDUFA target action date for August 22, 2026 as the deadline to […]
Santhera Pharmaceuticals announced results from the ongoing Guardian study in Duchenne, and showed that AGAMREE had comparable effectiveness compared to historical cohorts taking prednisone or deflazacort, based on time to […]
As an early investor in Dyne Therapeutics, CureDuchenne is happy to share that new analyses out to 24 months showed improvement in heart and lung function with z-rostudirsen compared to […]
As an early funder of Entrada Therapeutics, CureDuchenne is pleased to share that after the independent Data Monitoring Committee reviewed the safety data from Cohort 1 of the ELEVATE-44-201 trial […]
Sarepta Therapeutics announced positive topline data from the EMBARK trial, showing that Elevidys, 3 years post treatment, significantly slows disease progression on functional measures in ambulatory individuals who were 4-7 […]
