Australian National Spinal Muscular Atrophy Registry
The Australian National SMA Registry provides an opportunity for Australian SMA patients to participate in clinical trials being undertaken anywhere in the world. The Registry is linked directly to the TREAT-NMD global network of registries, an international effort which has proven effective in improving the health and management of boys with DMD. The Registry provides an important enabling tool for clinicians and clinical trial sponsors to quickly identify patients suitable for each study, particularly those therapeutic strategies that target specific genetic defects.
Potential Benefits of a SMA Registry
• Centralise information and provide an interface between patients, doctors and researchers
• Coordination of diagnosis, therapy and prevention – universally available across Australia
• Promote sense of community and belonging among affected patients and families and provide ready access to information concerning standards of care, research and available therapies
• Monitor and benchmark data to improve health care performance across institutions and providers
• Promote baseline standards of care available to all patients and families – provision of the “right care at the right time”
• Provide essential information necessary to leverage government and industrial support for further infrastructure
The database is open to anyone affected by DMD and is entirely voluntary – data obtained is held securely and confidentially. Most of the clinical and genetic information about each patient will be entered locally by the patient’s primary physician in association with the genetic testing laboratories in each state.