Patient Registry


the Patient Registry

 

 

 

This Patient Registry is a database of very basic patient information which will allow, when the time comes, efficient, fast tracked, local clinical trials of new treatments. The registry, the first of its kind for the congenital myopathies, takes the form of an electronic database that:


 
  • Encourages the development of more research.
  • Provides neuromuscular researchers with specific patient information to assist their research.
  • Helps with the organisation of new clinical trials.
  • Enhances clinicians' and other health professionals' ability to efficiently deliver treatments for this disease.

The registry is very straightforward and easy to complete within a short amount of time (about 20 minutes). The first step involves the patient completing a self-reported questionnaire and the second step involves them completing a consent form, which will tell us how the data can be used. Both steps are required to complete a patient's registration.

The registry has been set up by the Myotubular Trust, working in partnership with TREAT-NMD (Translational Research in Europe - Assessment and Treatment of Neuromuscular Diseases), a European neuromuscular network addressing the fragmentation currently hindering translational research for cutting edge therapies in rare neuromuscular diseases (NMD).


Dr Heinz Jungbluth, Senior Lecturer and Consultant Paediatric Neurologist in charge of the Neuromuscular Service at the Evelina Children’s Hospital, Guy's and St Thomas' NHS Foundation Trust, London has been liaising with other leading global academics in this field to agree the type of questions that are important for us to include on the registry and which will assist with their research.

We aim for this to be the most comprehensive global registry there is for myotubular and centronuclear myopathy.

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