Close
| Sponsor / Funder | Ultragenyx and Newcastle University |
| CI | Professor Hanns Lochmüller |
| Sites | Newcastle |
| Contact details | becky.davis@newcastle.ac.uk |
| More information |
|
This is a public-private partnership between Ultragenyx Pharmaceutical Inc. (USA) and Newcastle University (UK).
Primary objective
Tto improve knowledge and treatment development of GNE myopathy. This will be done by collecting data on clinical presentation and progression.
The program allows a combination of longitudinal data collected through:
- online global patient registry
- hospital based natural history study
The Natural History study is performed by selected centres in Europe, Middle East and North America.
Following approval from the Steering Committee and Ethical Committee, anonymous data from the registry will be available to:
- clinicians
- researchers
- patients
- families
- and patient organisations
This information could provide insight into the disease and help drive clinical trials. That could lead to better treatment strategies.