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WMR Trialling New Treatment for Rare Neuro Disease

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It’s a natural human trait to want to plan. It’s natural to want to know what is happening for your foreseeable future.

That’s one of the reasons being diagnosed with a rare disease like Pompe Disease has such an impact on a sufferer’s outlook and head space.

Late onset (or juvenile/adult) Pompe disease is the result of a partial deficiency of GAA. The onset can be as early as the first decade of childhood or as late as the sixth decade of adulthood. The primary symptom is muscle weakness progressing to respiratory weakness and death from respiratory failure after a course lasting several years.
Source: International Pompe Association

Wayne Brown was diagnosed with Pompe Disease last year. In spite of the rarity and severity of the disease, he has remained upbeat and positive. Part of his positive outlook has come from being able to participate in a worldwide study of a new treatment option.

Suddenly, as far as they can see, the future consists of symptom management, doctor’s appointments and the limited available treatment.

Every fortnight, Wayne travels from Kingaroy to Wesley Medical Research to be a part of a multicentre study being conducted in North and South America, Europe, Australia and South East Asia.

The purpose of this study is to evaluate the action and outcomes of a new study drug, compared to the currently available treatment, in the management of patients with late onset Pompe disease.

Clinical Nurse Jacqui Langton taking baseline measurements while Wayne Brown receives his infusion.

Approximately 96 participants will take part in the three year study, at approximately 27 sites in 25 countries. It is anticipated that there will be one or two participants in Australia. So Wayne Brown definitely sees his glass as half full.

“You can’t underestimate the psychological impact of being involved in a clinical study like this. To have such a rare disease, and to know that in worst case, my symptoms can be halted, or could possibly have a bounce back or remission of damage, does amazing things for my state of mind,” said Wayne.

Dr Robert Henderson, a neurologist at Wesley Hospital with a special interest in clinical trials for Motor Neurone Disease and neuromuscular disorders, diagnosed Wayne and identified him as an ideal candidate for this trial and applied immediately for his participation.

But the benefits aren’t limited to accessing world-leading treatment options for rare diseases. As Wayne tells it, “I also get to see the amazing group here at Wesley Medical Research every fortnight. They have made this a home away from home. An extended family who are extremely professional, but take such great care of me.”

When asked what he would like to say to potential donors, Wayne simply said, “Please donate to keep research projects like this alive. I am seeing the benefits beyond the medicine. I’m able to be part of something bigger, and you can too.”

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