A patient registry is one of the most efficient ways to quickly locate and recruit patients for clinical trials.
This is particularly vital for rarer conditions, like myotubular and centronuclear myopathy.
The registry was created by the Myotubular Trust in partnership with TREAT NMD and a number of leading neuromuscular researchers, as promising new therapeutic strategies are currently being developed for muscle diseases in general and most specifically for the myotubular and centronuclear myopathies (MTM/CNM). When the time is right these treatments will need to be tested in clinical trials.
The Myotubular and Centronuclear Myopathy Patient Registry will make the recruitment of MTM/CNM patients for clinical trials easier by helping to identify suitable patients for particular trials, and enable patients to be contacted and informed quickly when there is a trial they might be interested in. It can also be used to inform patients about new treatments that might be relevant to them. The registry will also assist with research; providing scientists with important information about the prevalence and course of myotubular and centronuclear myopathy.