Brogidirsen and the Promise of Exon 44 Skipping: New Horizons in RNA Therapy for Duchenne Muscular Dystrophy – Patient Worthy

First participant dosed in novel AAV-gene therapy trial for DMD-associated cardiomyopathy

Medera, and its clinical division Sardocor, announced that the first participant was dosed with AAV1.SERCA2a in a Phase 1b clinical trial for cardiomyopathy secondary to Duchenne.Cardiac complications are the leading cause of mortality in Duchenne, and this experimental gene therapy delivers the SERCA2a gene with the goal of correcting the calcium dysregulation in cardiac muscle that leads to progressive fibrosis and heart failure.  This experim…

Brogidirsen and the Promise of Exon 44 Skipping: New Horizons in RNA Therapy for Duchenne Muscular Dystrophy – Patient Worthy

Duchenne muscular dystrophy (DMD) is a devastating, inherited muscle-wasting disorder caused by mutations in the DMD gene, leading to an absence of functional dystrophin protein. While current standards of care, such as corticosteroids and supportive therapies, have extended patient lifespans and improved quality of life, they remain largely palliative and do not address the root […]