PatientCrossroads CMD


Ongoing Studies

The CMDIR’s mission is to provide the latest information regarding international CMD clinical studies and trials and connect the CMD community: people with CMD, their families, doctors, researchers and the pharmaceutical industry. The CMDIR Advisory Board reviews all clinical studies posted. 

1 Prospective, Longitudinal Study of the Natural History and Functional Status of Patients With Myotubular Myopathy (MTM)
2 Use of Tyrosine in Nemaline Myopathy
3 A Study to Test Hyperinsufflation to Slow the Rate of Decline in Children With Collagen VI -CMD
4 Development of a Proxy Motor Outcome Assessment in Young Children with Neuromuscular Disease
5 Clinical Trial Readiness for the Dystroglycanopathies
6 MTM Genetic Testing Study
7 Prospective Event Rate Study in Males with X-linked Myotubular Myopathy
8 Clinical Trials and Studies Overview