The Jennifer Trust has joined forces with TREAT-NMD to create a comprehensive SMA Patient Registry in the UK.
Scientific advances over recent years have lead to substantial changes in the treatment of many diseases. New therapeutic strategies are being developed and, for some of these treatments, plans for large studies involving patients from more than one country are already in place.
Several new therapeutic strategies for neuromuscular diseases like SMA target specific genetic defects. When a clinical trial is being planned, it is very important that patients suitable for that trial can be found and contacted quickly. The best way of ensuring this can happen is to make sure that patients' details are all collected together in a single database or "registry" that contains all the information that researchers will need, including each patient's particular genetic defect and other key information about their disease.
TREAT-NMD is an international initiative bringing together some of the world's leading neuromuscular specialists in a pan-European 'network of excellence' aimed at improving treatment and finding cures for patients with neuromuscular disorders.
The network's goals include setting up an international clinical trials coordination centre that will provide advice on how to conduct trials of the highest standard, establishing international patient databases and biobanks that will supply researchers with the resources they need to develop cutting-edge therapies, and making available top-level training for network members.
The TREAT-NMD initiative is being coordinated by Professor Kate Bushby and the UK SMA database is being managed by Professor Hans Lochmuller. Both are based at Newcastle University.
Late last year the Trust worked with the TREAT-NMD team to develop a pilot questionnaire that was sent to 100 of our members. The responses from that pilot were used to fine tune the final online questionnaire that was launched at the Trust's Annual Conference on 5th July
The comprehensive database will be held and managed in strict confidence by the TREAT-NMD team and no one at the Jennifer Trust will see the individual responses.
The completed database will provide researchers and doctors with an important resource when clinical trials are being designed, and those people with SMA on the database will stand the best chance of being able to participate in those trials.
We hope that everyone who has SMA or cares for someone with the condition will add their details to the register.
To access the online registration form, please visit this link. | 
