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 CALLISTO Omigapil Study (Collagen VI & LAMA2)
2 Congenital Muscle Disease HYPOGLYEMIA Survey (CMD Hypoglycemia)
3 MTM1 Carrier Survey
4 Prospective, Longitudinal Study of the Natural History and Functional Status of Patients With Myotubular Myopathy (MTM)
5 Use of Tyrosine in Nemaline Myopathy
6 A Study to Test Hyperinsufflation to Slow the Rate of Decline in Children With Collagen VI or LAMA2 -CMD
7 Development of a Proxy Motor Outcome Assessment in Young Children with Neuromuscular Disease
8 Clinical Trial Readiness for the Dystroglycanopathies
9 MTM Genetic Testing Study
10 Prospective Event Rate Study in Males with X-linked Myotubular Myopathy
11 Clinical Trials and Studies Overview