This is a multi-centre, prospective, cross-sectional and longitudinal natural history study in NDM patients.
|Sponsor||University College London|
|Funder||National Institutes of Health (NIH - USA)|
|CI||Professor Michael Hanna|
|Sites||London Queen Square|
To collect standardised data from NDM patients, to include:
- clinical symptoms
- exam findings
- results of strength, functional, and electrophysiological testing
Genetic testing will allow identification of individual NDM subtypes. Appropriate endpoints will then be identified and used in studies of potential treatments.
20 patients were enrolled at the National Hospital for Neurology and Neurosurgery.