Launch of National Duchenne Muscular Dystrophy Register
On Monday November 15th 2010, the Director General of Health launched the Duchenne Muscular Dystrophy (DMD) Registry. Patient registries are infrastructure that supports clinical and translational research.
The DMD registry was created in response to developments in genetic technology and in particular clinical trials currently being planned, that offer the hope of treatments for this devastating disease. These treatments will extend the existing management options, including physical and clinical interventions, and community support, and potentially give patients further improvements to their quality of life.
The Registry collates the patient’s clinical and genetic mutation data and will accelerate the recruitment process for Australian DMD patients into international multicentre clinical trials. To facilitate the recruitment process the Registry links into the TREAT-NMD (Translational Research in Europe for the Assessment and Treatment of Neuromuscular Disease) global network of registries.
The Australian National DMD Registry was developed by the Office of Population Health Genomics, with support from other State clinical and genetic testing services, the Centre for Comparative Genomics at Murdoch University and patient support organisations. Further neuromuscular and rare diseases registries are planned for Australia.
Prof Monique Ryan, Director Neurology RCH and Boris M Struk, Executive Director MDA together with representatives from other States including NZ, are on the NMD Advisory Committee. This committee meets regularly via teleconference, oversees the development of the registry and approves applications from research institutions from around the globe.
The NMD Registry is an overarching structure to house clinically relevant disease specific registries for conditions such as spinal muscular atrophy (SMA) and myotonic dystrophy (DM).
In December 2015, the OPHG launched the myotonic dystrophy registries (DM) following its development. The launch of the DM registries will link patients with international clinical trials through the TREAT-NMD global network.
MDA is proud to be part of the team associated with the development of the DMD Registry and in the knowledge that our collective inputs will ultimately make a difference.