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 […]
As an early investor in Entrada Therapeutics, we are pleased to share that in 2026 Entrada plans to have four clinical-stage programs in Duchenne – for individuals amenable to skipping […]
CureDuchenne applauds the U.S. Department of Health and Human Services (HHS) for adding Duchenne muscular dystrophy to the Recommended Uniform Screening Panel (RUSP) for newborn screening. We also extend our congratulations to Parent Project […]
