Solid Biosciences Reports Positive Initial Clinical Data from Next-Generation Duchenne Gene Therapy Candidate SGT-003 Solid Biosciences, announced positive initial results from INSPIRE DUCHENNE, their next-generation gene therapy clinical study of […]
Cumberland Pharmaceuticals announced positive cardiac results from Phase 2 trial in Duchenne Cumberland Pharmaceuticals released positive top-line results from its Phase 2 FIGHT DMD trial, a 12-month placebo controlled study […]
As an early funder of Entrada, CureDuchenne is pleased to share that Entrada received authorization in the United Kingdom (UK) to start a Phase 1/2 trial for individuals with Duchenne […]
Upcoming Events Offer Treatment Updates and Essential Resources to Support Families and Enhance Quality of Life NEWPORT BEACH, Calif., January 31, 2025 – CureDuchenne, a global leader in advancing research and […]
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 […]