Sarepta Therapeutics shared positive topline results from Part 2 of the EMBARK study, showing that treatment with the microdystrophin gene therapy, Elevidys, is associated with sustained benefits and disease stabilization […]
By Debra Miller, Founder and CEO of CureDuchenne Duchenne International Outreach: Bridging Gaps in Duchenne Care At CureDuchenne, we have always believed in the transformative power of connection—the kind of […]
Amenable to skipping exon 44 Avidity Biosciences, which received early funding from CureDuchenne, plans to submit for US FDA Accelerated Approval at the end of 2025 for Delpacibart zotadirsen (del-zota), […]
Dyne Therapeutics, which received early funding from CureDuchenne, plans to pursue US Accelerated Approval from the FDA in 2026 for DYNE-251, an exon-skipping experimental therapeutic for individuals with Duchenne amenable […]
Avidity Biosciences, which received early funding from CureDuchenne, plans to submit for US FDA Accelerated Approval at the end of 2025 for Delpacibart zotadirsen (del-zota), an exon-skipping experimental therapeutic for […]
Capricor Therapeutics, which received early funding from CureDuchenne, has completed a Biologics License Application (BLA) with the US FDA, seeking full approval of deramiocel for individuals with Duchenne cardiomyopathy. Deramiocel […]
Australian company Percheron Therapeutics released topline 6-month data from its Phase 2 study of avicursen in non-ambulatory individuals with Duchenne. The trial did not meet its primary endpoint, and there were […]
As an early funder of Edgewise, CureDuchenne is pleased to share that the Phase 2 Trial of sevasemten in Becker muscular dystrophy met its primary endpoint of reduction in creatine […]
PepGen has received a clinical hold notice from the FDA regarding its Investigational New Drug (IND) application in Duchenne amenable to skipping exon 51, and will be unable to initiate […]
