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"It is our duty as medical researchers to find a cure"

Speechless for Rett Syndrome would like to introduce you to Dr. Wendy Gold.

Dr. Gold has been studying Rett syndrome for the past 10 years, and is leading the Rett Syndrome research being undertaken at the Kids Neuroscience Centre, Sydney Children’s Hospitals Network.

What brought you to working with Rett Syndrome?

My father was a paediatrician and mother a paediatric nurse and they instilled in me the importance of helping sick kids. I have always realised the value of medical research and how new therapies can be developed if enough energy and determination are poured into areas of critical need.

Rare neurological disorders like Rett syndrome fall into this category and it is our duty as medical researchers, to find a cure.

What has impressed you most about the Rett Syndrome community in Australia?

The Rett community in Australia is very unique and an should be an inspiration to akin communities. There are only about 400 girls with Rett in Australia making it very challenging for a community to stay cohesive. However, despite this the families rally together and support each other. The Rett community in Australia is a flagship for other rare disease communities and should be applauded.

What would having a cure for Rett mean ?

Finding a cure for Rett opens up many therapeutic windows for other neurological disorders. If we can prevent developmental delay, enabling girls to talk and walk and be seizure free, we will give hope not only to the Rett community but to other disorders such as CDKL5 deficiency disorder and other disorders where seizures and related neurological symptoms are prominent features.

Is there something that you are currently doing in the lab that you are particularly excited about?

Yes! We are developing gene therapies for Rett. The disorder is very complex where MECP2 is known as a Goldilocks gene. What this means is we can’t have too much or too little in our body and the amount has to be ‘just right’. We are therefore testing a number of different strategies to ensure we have an effective and safe gene therapy to offer the Rett syndrome community.

Do you have a message for the Rett Syndrome Community?

Thank you for all your support. I’m always available to answer any questions and of course, discuss ways in which to fundraise for research. A cure is possible, but of course researchers are always in need of more funding to support our work.

This month, Dr. Gold and her team are wearing purple lab coats to raise awareness for Rett Syndrome! We think they look very fetching indeed!

You can read about Dr Wendy Gold's research on the Rett Research Australia website

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