New Treatments for Duchenne Muscular Dystrophy: Emma Ciafaloni, MD The professor of Neurology and Pediatrics at University of Rochester Medical Center discusses the new and future treatments for Duchenne muscular dystrophy. [WATCH TIME: 4 minutes]
WATCH TIME: 4 minutes
“These are all strategies that are really advancing very rapidly. I think that the field is now really moving much faster than in the past because of the collaboration between pharma and academia, and the patients and the families.”
Currently, all of the therapeutic strategies to restore dystrophin in patients with Duchenne muscular dystrophy (DMD) involve semifunctional, biochemically abnormal types of dystrophins and are not the full-length protein. The goal for these treatment strategies is to optimize minidystrophins or microdystrophins small enough to be packaged, retaining the protein function. Future treatments for DMD need to improve vectors such as having a lower delivered dose, less liver toxicity, and greater muscle and cardiac transduction.
At the 2022 American Association of Neuromuscular and Electrodiagnostic Medicine (AANEM) annual meeting, September 21-24, in Nashville, Tennessee, Emma Ciafaloni, MD, FAAN, professor of neurology and pediatrics, University of Rochester Medical Center, presented a talk on the new treatments for DMD. She explained that future research on the treatments should improve their clinical trial design and outcome measures, and reduce the time and number of patients needed to reach approval for the therapies. Specifically, there needs to be a development of sensitive outcome measures for younger patients and older nonambulatory patients, to help with earlier diagnosis. For the future care of patients with DMD, having the next generation of neuromuscular neurologists trained will benefit patients in giving them higher quality care.
In a recent conversation with NeurologyLive®, Emma Ciafaloni, MD, discussed the progressing research that is occurring in the field of DMD and the therapeutic strategies that are being developed more rapidly. She also spoke about treatments that are more geared toward the downstream pathology of muscle degeneration like fibrosis, inflammation, and regeneration.