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Stepping up in the global fight

Stepping up in the global fight to find a treatment for devastating childhood diseases

Children and young people with Duchenne muscular dystrophy (DMD) have increased access to lifesaving treatments through clinical trials as a result of the New Zealand Duchenne muscular dystrophy Registry newly established by the Muscular Dystrophy Association of NZ.

The Registry Curator and MDA’s Membership Services Manager Miriam Rodrigues said “The DMD registry initiative was driven by MDA members in response to recent developments in genetic technology, and in particular clinical trials currently being planned, that offer the hope of treatments for this devastating disease”.

Today’s Australasian-wide launch represents the culmination of a unique collaboration between patients (and those with affected family members), clinicians and clinical trial sponsors to step up the global fight to find a treatment for this devastating disease. This collaboration does not end with the establishment of the Registry – it will continue on through the long term commitment of the Muscular Dystrophy Association of NZ to continue to promote the opportunity for patients wanting to participate in trials, of clinicians who keep their clinical information current and provide information to their patients about the existence of new trials or treatment options, and finally, of clinical trials sponsors to make feasibility enquiries of the Registry and to notify clinicians, through the Registry, of new studies.

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The Registry, collates the patient’s clinical and genetic mutation data to improve the care of DMD patients, and to accelerate the recruitment process for NZ DMD patients into international multicentre clinical trials. The Registry links into the TREAT-NMD global network of registries, opening up opportunities for NZ DMD patients to participate in clinical trials being undertaken anywhere in the world.

The TREAT-NMD global network of national registries has proven effective in improving the health and management of boys with DMD. The next step is to launch registries that will enable rapid access for trials of other neuromuscular and rare diseases such as spinal muscular atrophy (SMA) and myotonic dystrophy.

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