75 mg/ml gocce orali clinical trials

Deflazacort TREATment in LMNArelated congenital muscular dystrophy: study of clinical effectiveness and search for reliable biomarkers

This study is testing a medicine called Deflazacort drops for people with muscle weakness caused by a condition called laminopathy. Researchers want to see if the medicine helps improve muscle strength and if there are ways to measure its effects over time.