New muscular dystrophy registry to provide hope for thousands

Young Western Australians with Duchenne muscular dystrophy (DMD) will have better access to worldwide clinical trials of new therapies and treatments thanks to a new national registry.

Health Director General Kim Snowball said the Australian National DMD Registry would help researchers quickly identify patients who may be suitable for a range of clinical trials.

"Whenever a clinical trial is being planned, it is vital that patients suitable for that trial can be found and contacted quickly," Mr Snowball said.

"This registry will collate patient’s clinical and genetic mutation data so researchers have instant access to important information such as each patient’s particular genetic defect and their recent medical history.

"Signing up to the registry is voluntary and all information will be kept confidential and stored securely. Eligible patients will be informed about new trials and studies through their primary physicians."

The Australian National DMD Registry was developed by WA Health’s Public Health Division, with support from other State clinical and genetic testing services, the Centre for Comparative Genomics at Murdoch University and patient support organisations.

Duchenne Muscular Dystrophy affects mainly young boys and is characterised by progressive muscle weakness caused by an absence of dystrophin – a protein that helps keep muscle cells intact.

Patients with the condition are often affected by a lifelong disability and require high levels of family, social and medical care.

Mr Snowball said despite the debilitating nature of the condition, recent scientific advances had led to improved treatment methods and patient care.

"It is important that patients with DMD are kept informed of any medical developments so they can take advantage of new treatments as they become available," Mr Snowball said.

"This registry will open up opportunities for patients to participate in clinical trials not only in Australia but also around the world.

"By feeding into a global network of National DMD registries from countries including, France, Italy, Germany, Spain, and the United Kingdom, eligible Western Australians will be able to receive notification if they are suitable for a clinical trial being undertaken from outside Australia."

Mr Snowball said most of the clinical and genetic information about each patient will be entered by their local clinical service in association with the genetic testing laboratories in each state.

(Source: Government of Western Australia, Department of Health)