The map to develop a life-changing genetic treatment for Prader-Willi syndrome

By Prader-Willi Research Foundation Australia

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A little bit of context

Prader-Willi syndrome is a genetic condition that is cruel and devastating to both the person who has it and their family. Life expectancy is short (averaging 35 years) and in the interim is unimaginably difficult. People with PWS have physical and intellectual disability and enormous medical complications.

They can have a constant sense of starvation, mental illness (severe anxiety, bipolar, psychotic events), debilitating physical fatigue, severe spinal curvature, poor emotional control (including daily rages akin to those in autism), gut dysfunction, impaired thirst, temperature and pain mechanisms and too many medical complications to list. Every day is hard, hard work.

More than 90% of siblings have post-traumatic stress disorder from living with PWS. Families have more impaired wellbeing than any other developmental disability (University of Sydney).

However today's science offers us unprecedented opportunities to develop life-transforming treatments and the Prader-Willi Research Foundation Australia was established to make these treatments a reality. Scientists advise us that we can now target the underlying genetics of PWS and that it is likely that the brain will catch up on development.

But there’s a problem

Right now, developing the right biotech is like driving from Sydney to Perth without a map. Which road is best, safest, fastest? There are a number of different biotechnologies in which we could invest but we don't know which are the best to target the PWS genes. We also don't know how easy or fast it will be to make them safely available to patients.

Here’s what we’re doing about it

We will draw a map. We will thoroughly evaluate the available biotechnologies to determine which ones offer the best chance of a treatment, safely and quickly. This way we can raise precious funds for the development phase and give donors confidence that their money will be spent in the best possible way.

You can join us

Our report will cost $30,000. It will enable us to raise funds to develop the treatment and give us the best chance of changing lives as quickly as possible.

And here’s some amazing perks for supporting us that you can’t live without

You are supporting a small foundation that is dedicated to changing lives that most of us could not even imagine. There are around 1500 families in Australia affected by PWS and yet this condition remains largely hidden. Only 2 research projects are currently happening in Australia. Families have high levels of social and economic exclusion as well as high levels of anxiety and other psychological conditions (including PTSD) due to the challenge of caring for someone with PWS. You can help us change this.

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Team Members

Kathlene Jones