Sarepta Therapeutics has shared that a young man with Duchenne passed away following treatment with ELEVIDYS, an AAV-delivered micro-dystrophin gene therapy. Sarepta has shared that the individual suffered from acute […]
2025 MDA Conference Updates We’re at the 2025 MDA Clinical & Scientific Conference this week and are pleased to share research updates from companies presenting on the latest advancements in Duchenne muscular […]
Capricor Therapeutics received early funding from CureDuchenne, and today Capricor released new data reporting that individuals treated with deramiocel over three years experienced an average decline in Performance of the […]
CureDuchenne provided early funding to Dyne Therapeutics, and is pleased to share that Dyne has released new long-term data from the DELIVER trial in individuals amenable to skipping Exon 51. […]
CureDuchenne provided early funding to Avidity Biosciences, and are pleased to share they have released positive topline data from the EXPLORE44 trial in individuals amenable to skipping exon 44. Data […]
PepGen, which received early funding from CureDuchenne, announced that they are temporarily pausing their CONNECT2 study in the UK in order to focus resources and first review data from the […]
CureDuchenne provided early funding to Edgewise Therapeutics, and we are pleased with their progress as recently shared in their fourth quarter and full year 2024 reports. In Becker muscular dystrophy, […]
CureDuchenne provided early funding to Capricor Therapeutics, and we are pleased to share that the FDA has accepted its Biologics Licenses Application (BLA) seeking approval for Deramiocel to treat cardiomyopathy in […]
CureDuchenne was an early funder of Avidity, and today we share an important milestone: Enrollment is now complete for the EXPLORE44-OLE clinical trial. Avidity is pursuing accelerated approval for del-zota […]
