Creatine/Creatinine Ratio and Myostatin as Biomarkers to Monitor Muscle Function in Duchenne Muscular Dystrophy Patients - PubMed
4 hours ago
- #muscle function monitoring
- #clinical trial design
- #DMD biomarkers
- The study investigates myostatin and creatine/creatinine ratio (Cr/Crn) as biomarkers to monitor muscle function in Duchenne Muscular Dystrophy (DMD).
- Lower Cr/Crn and higher myostatin levels are associated with better functional performance, such as higher scores on the North Star Ambulatory Assessment (NSAA) and Performance of Upper Limb (PUL2.0), and a reduced risk of losing ambulation.
- Incorporating these biomarkers as endpoints in clinical trials could reduce required sample sizes by more than half due to their lower intra-patient variability compared to traditional functional tests.
- Findings support using myostatin and Cr/Crn to enhance trial design and endpoints in DMD clinical and interventional studies.