The DEM-CHILD registry project, led by Angela Schulz, MD, of Germany’s Children’s Hospital and University Medical Center Hamburg-Eppendorf, aims to collect the world’s largest, clinically and genetically best characterized set of NCL patients.
The map shows the current partners, who are working together to develop and expand the project worldwide. Patient Organisations are an integral part of the consortium and representatives from the BDFA attend the annual meetings.
Why are registries important?
Having accurate and up to date information is vital for the successful clinical trials and for the development of potential therapies but there are other benefits that can often be overlooked.
Understanding and building up a picture of what is happening, in the disease, can assist in proactive interventions for patients, enabling the delivery of better supportive care and so improving quality of life.
By studying current data indicators of early disease can be identified to assist in looking for improved and earlier methods of diagnosis.
How is the BDFA involved?
The BDFA represents the patient voice, bringing to the discussion our experience of how many families wish to take part in the study, the challenges of patient involvement, including the issues that were highlighted at the registry session at the BDFA Family Conference last October.
We are also very proud to be funding partners in this work, with the BSDRA, Hope for Bridget and Noah’s Hope. We have funded a researcher, Dr Susanne Lezius, based with Dr. Angela Schulz (Project coordinator) in Hamburg. This will facilitate faster interpretation of the data collected so far making a significant difference to our current working knowledge of Batten disease and provide vital information for potential future clinical trial development.
How can you help?
If you would like to take part in the registry, or receive more information on what is involved, please contact Dr. Ruth Williams, who is the lead clinician for the UK.
0207 188 3998 or ruth.williams@gstt.nhs.uk
