Publications by authors named "Laura Ambrosoli"
Front Neurol
January 2023
Article Synopsis
- The study aimed to evaluate the effectiveness and safety of givinostat, a treatment for Becker muscular dystrophy (BMD), in adult males, comparing it to a placebo over 12 months.
- Out of 51 enrolled patients, the treatment did not show a statistically significant improvement in total fibrosis or other primary endpoints after 12 months, although secondary MRI assessments hinted that givinostat might slow disease progression.
- Most adverse events reported were mild to moderate, with 88.2% of patients on givinostat and 52.9% on placebo experiencing side effects.
Article Synopsis
- Becker muscular dystrophy (BMD) is a condition with varying severity, and this study aimed to identify biomarkers to aid in clinical trials by analyzing a cohort of affected patients.
- The study involved 51 ambulatory male patients aged 18 to 65, examining muscle function, strength, and using MRI to assess fat content and muscle tissue characteristics.
- Results showed significant correlations between muscle tissue health, MRI findings, and functional performance, indicating that MRI fat fraction and histological data may serve as useful measures in future BMD research, though further validation is needed.
