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 […]
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 […]
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 […]
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 […]
By Debra Miller, Founder and CEO of CureDuchenne I’m honored to share two transformative events that embody both the resilience of the human spirit and the strength of our community: […]
CureDuchenne is pleased to share that PTC Therapeutics has announced that the FDA has accepted for review the New Drug Application (NDA) for Translarna (ataluren) for the treatment of Duchenne […]
Read a message from our founder and CEO The recently released award-winning Netflix documentary “The Remarkable Life of Ibelin,”* highlights the life of Mats Steen, a Norwegian World of Warcraft […]
