Stay updated with breaking news from Admd. Get real-time updates on events, politics, business, and more. Visit us for reliable news and exclusive interviews.
A recent study encapsulates how patients and caregivers characterize the impact of Duchenne muscular dystrophy (DMD) on patients’ physical limitations and symptom burden, potentially helping to inform patient-centered strategies for assessing clinical outcomes in DMD research.
This year’s top content in the Duchenne muscular dystrophy (DMD) space focused on a novel gene therapy approval, the high cost of care for this genetic disorder, and steps forward in other treatments.
Givinostat, a histone deacetylase inhibitor from Italfarmaco, was supported by data from the phase 3 EPIDYS study, a randomized, double-blind, placebo-controlled, multicenter trial investigating the agent vs placebo.
Investigators evaluated longitudinal MRI and spectroscopy outcomes and ambulatory function among 180 patients with Duchenne muscular dystrophy (DMD) to establish the utility and reproducibility of magnetic resonance measures of muscle quality at different disease stages.
For This Year in Medicine, CGTLive looks back at a landmark first approval, the new treatment opportunities it has provided, and the unmet needs in the field it has brought to light.