Clinical TrialsThe 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. Clinical trial information is pulled directly from clinicaltrials.gov using CMD and LGMD nomenclature below.
What is a clinical study? A clinical study answers questions about CMD. A study may enroll people with CMD or may access information retrospectively (in the past) through chart review.
How does a clinical study differ from a clinical trial? A clinical study may not test an intervention, yet answers important questions about CMD. Clinical studies can lead to clinical trials.
What is a clinical trial? A clinical trial measures an intervention. An intervention can either be a medical compound (drug) or a treatment plan (exercise regimen).
Clinical study and trial information is posted for the following CMD/LGMD subtypes and associated genes:
CMD Subtypes included in the CMDIR:
- Ullrich CMD
- Merosin deficient (MDC1A)
- CMD, undiagnosed (including merosin positive)
- Dystroglycanopathy (WWS, MEB, Fukuyama)
- Integrin alpha 7 deficiency
- Integrin alpha 9 deficiency
- Laminopathy (Lamin A/C)
- SEPN 1 related myopathies: SEPN1 (selenoprotein deficiency) and RYR 1 (ryanodine receptor)
LGMD subtypes included in CMDIR:
- Bethlem myopathy
- Dystroglycanopathies (LGMD2K, LGMD2I, LGMD2L, LGMD2N)
If you are an investigator with a CMD clinical study or trial that you would like to submit to the CMDIR Advisory Board to post on the CMDIR site, please contact This e-mail address is being protected from spambots. You need JavaScript enabled to view it
