Results from the Phase 3 EMBARK Clinical Trial for DMD скачать в хорошем качестве

Results from the Phase 3 EMBARK Clinical Trial for DMD

Stefanie Mason, MD, Senior Medical Director and Clinical Development Lead for Duchenne muscular dystrophy (DMD) at Sarepta Therapeutics, discusses results from the phase 3 EMBARK clinical trial for DMD. DMD is a rare genetic disorder due to mutations in the dystrophin gene that leads to progressive muscle wasting. DMD occurs primarily in males, though in rare cases may affect females. The symptoms of DMD include progressive atrophy of both skeletal and heart muscle. Early signs may include delayed ability to sit, stand, or walk and difficulties learning to speak. Muscle weakness is usually noticeable in early childhood. DMD is caused by genetic mutations in the DMD gene. Elevidys (delandistrogene moxeparvovec) is an rAAVrh74 vector-based gene therapy that delivers a functional form of the dystrophin gene. The EMBARK clinical trial is a phase 3, two-part, multinational, randomized, double-blind, placebo-controlled study assessing the safety and efficacy of delandistrogene moxeparovovec in patients with DMD ages 4 to less than 8 years. The safety profile observed was consistent with prior studies with no new safety concerns. Delandistrogene moxeparvovec did not illustrate a statistically significant difference in the primary endpoint of change from baseline to Week 52 versus placebo in The North Star ambulatory assessment (NSAA) total score. However, the treatment performed well in key secondary and other functional endpoints. The GST also supported the evidence of presence of functional treatment effect Additionally, a post hoc analysis of time to rise (TTR) showed TTR greater than 5 seconds in a larger portion of patients in the placebo group as compared to those treated with delandistrogene moxeparvovec. Chapters: The Current DMD Space 00:00 ELEVIDYS Overview 2:20 EMBARK Clinical Trial 3:59 Next Steps 6:55 Message to Physicians 7:51