About this Registry

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 myopathies.

The Myotubular and Centronuclear Myopathy Patient Registry was created in 2013 by the Myotubular Trust in partnership with TREAT-NMD and a number of leading neuromuscular researchers, prompted by the fact that promising new therapeutic strategies were being developed for muscle diseases in general and most specifically for the myotubular and centronuclear myopathies (MTM & CNM). When the time is right, any new treatments 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 enabling 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 also assists with research; providing scientists with important information about the prevalence and course of myotubular and centronuclear myopathy.

Newcastle University owns and independently operates this international registry as part of the global TREAT-NMD Neuromuscular Network. More information about patient registries is available on the TREAT-NMD website.

The registry is co-funded by the Myotubular Trust, Muscular Dystrophy UK, MTM-CNM Family Connection, and Astellas Pharma Inc.