Prader-Willi Syndrome Association of Queensland (Australia) Inc | Non-profit organisation

Prader-Willi Syndrome Association of Queensland (Australia) Inc

Phone: +61 423 529 835

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25.01.2022 Great catchup today with some new and familiar faces. Looking forward to another get together in the next few weeks. Watch this space!! Lisa

13.01.2022 **Link to the webinar with Professor Marnie Blewitt from 28 October 2020 now available** http://praderwilli.org.au/blog On 28 October many of our community tune...d in to hear Professor Marnie Blewitt from WEHI talk about her project to identify chemicals that activate one of the two major (silenced) PWS gene clusters, Magel2. A few years ago, Professor Blewitt stumbled across a chemical that silences an enzyme Schmd1 and as a consequence activates the Magel2 gene cluster i.e. a potential medicine for Prader-Willi. Because she had previously been introduced to PWRFA, Marnie was interested in investigating whether her accidental discovery could be turned into a therapy for Prader-Willi. We initially seed funded her $25,000 to validate her finding and she has then taken her work on to proof of concept of a gene therapy and developed a first chemical candidate for a pharma company to develop into a drug. Pharma companies need more than one chemical candidate to successfully develop a drug, so Professor Blewitt’s work has now been fast-tracked to the National Drug Discovery Centre in the search for more compounds that will silence Schmd1 and activate the silent Magel2 gene cluster. There’s still a way to go and no guarantees but this world-leading research offers so much hope for a medicine for our children. Be assured that all of this happened because of Prader-Willi Research Foundation Australia. We are the only Australian purpose-built organisation to grow research into life-changing treatments for PWS. We work to identify the most promising science to create real impact in our children’s lives. Our Research Director and Scientific Advisory Council ensures that projects we fund are robust scientifically. We work to attract the most gifted scientists to focus their efforts on PWS. We work to raise awareness amongst key decision-makers to support PWS. The only thing holding us back is funding. Please sign up via our website and you will be sent a link to the recording http://praderwilli.org.au/blog