Background Vamorolone is a dissociative steroidal anti-inflammatory drug that seeks to retain efficacy and reduce safety concerns in patients with Duchenne Muscular Dystrophy compared to […]
Recently, exon skipping therapy has been attracting attention for patients with Duchenne muscular dystrophy (DMD). This therapy uses a synthesized antisense oligonucleotide designed to skip […]
Background Vamorolone is a dissociative steroidal anti-inflammatory drug that seeks to retain efficacy and reduce safety concerns in patients with DMD compared to corticosteroids via […]
Objective: We report 2-year treatment period of vamorolone (VBP15-LTE) versus corticosteroid treatment data from the NorthStar UK (NSUK) Clinical Network and the CINRG Duchenne Natural […]
Background: Pharmacokinetic/pharmacodynamic (PK/PD) properties of exon-skipping therapies for DMD are traditionally evaluated by invasive muscle biopsies limiting longitudinal study of individual patients. Skin biopsies may […]
Background: Delandistrogene moxeparvovec (SRP-9001) is an investigational gene transfer therapy developed for targeted skeletal and cardiac muscle expression of micro-dystrophin, a shortened, functional dystrophin protein. […]
ATL1102, an antisense drug to CD49d, the alpha chain of VLA-4, has been evaluated in a Phase II study in nine non-ambulant patients with Duchenne […]
Background: Cardiomyopathy is a principal cause of morbidity and mortality in Duchenne muscular dystrophy (DMD). The development of therapeutics to treat cardiac complications is hampered […]
Duchenne muscular dystrophy is typically diagnosed between 4-5 years. A small percentage of boys are diagnosed in infancy, typically because of family history or incidental […]