- authorisedTherapeutic exploratory (Phase II)
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.
Italy - recruiting
Observatoire Des Patients Atteints de Laminopathies et Emerinopathies (Observatory for PAtients With Laminopathies and Emerinopathies)
This study is collecting information about people in France with rare genetic conditions called laminopathies and emerinopathies. It aims to understand these conditions better, including how they affect the body over time and what might predict their progression. The goal is to help find better ways to manage and treat these diseases.
France