Dynacure today announced its participation in the ‘Natural History Study and Functional Status of Patients with Myotubular Myopathy and other Centronuclear Myopathies’.
This prospective non-interventional longitudinal study investigates the natural history and function of 60 patients with MTM and other CNM from Europe, United States and Canada. The study was started in 2014 by the Institute of Myology, Genethon and Valerion Therapeutics to collect data on patients with the MTM1 gene (XLCNM). Dynacure will continue the study with the Institute of Myology (its sponsor) and extend it for 24 months. In addition to XLCNM, other subtypes of CNM patients resulting from a mutation in BIN1 and DNM2 will be enrolled.
The study objective is to follow-up the disease course, changes in disease severity and any progression of symptoms. Study specific functional assessments and patient questionnaires will be used, and will be based on age and ambulatory status of the participant.