This article first appeared in the Information Point newsletter Our World in 2018, when Jo Bullivant, curator of the MTM-CNM Patient Registry wrote about the registry.
Have you registered in the Myotubular and Centronuclear Myopathy (MTM and CNM) Patient Registry yet? The MTM and CNM Patient Registry is an international registry open to MTM or CNM patients and female carriers of x-linked myotubular myopathy (XLMTM). It is funded by the Myotubular Trust and Muscular Dystrophy UK and managed by the John Walton Muscular Dystrophy Research Centre at Newcastle University, as part of the TREAT-NMD Alliance.
There are many reasons why patient registries are so important in rare diseases, including:
- they provide doctors with information about the conditions, to help improve standards of care
- they support and encourage research by providing (anonymous) data about the conditions
- being part of a registry means that researchers or pharmaceutical companies can ask the Registry Curator to let participants know if there is a trial or research study that they might be eligible for
- pharmaceutical companies and other organisations are increasingly acknowledging the value of real-world, patient-reported data, as it can potentially reduce the need for placebo arms in clinical trials and help to develop patient-centred outcome measures.
Being part of a registry will also mean that researchers or pharmaceutical companies can ask the Registry Curator to let you know if there is a trial or research study coming up that you might be eligible for.
Learn more about the Myotubular and Centronuclear Myopathy Patient Registry
To learn more about the Patient Registry and to register online, visit the website or email Jo Bullivant, the Registry Curator with any questions.
- The Myotubular and Centronuclear Myopathy Patient Registry website
(registration is available in English, German, Spanish, Polish, Italian and French).
- Email: firstname.lastname@example.org
Information about clinical trials can also be found on Patient Registry website.
Real world data in clinical trials
If you would like to find out more about the use of real world data in clinical trials, take a look at the article from Muscular Dystrophy News below.