<div><strong>Twenty-one children. A rare disease. A real chance. </strong>
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<div>Sophia Ibrahim (9), Emil (7), Mary (13) and Neveah (10), Jonathan (9), Charnice (4) and Madison (3) – five lovely and joyful kids from Australia, the United States, South Africa, UK and Germany. What do they have in common? A very rare genetic disorder: PGAP1-CDG.
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<div>They can’t speak, walking is difficult or impossible for some, and many things are only possible with support. And yet – they laugh, play, cuddle – and their families fight for them every single day.
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<div>“Worldwide, only 21 children are known to be affected by this gene defect,” say the geneticists, “with odds of around 1 in 21 million.”
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<div><strong>But now, there is hope.</strong>
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<div><strong>A Breakthrough in Sight – We Need You!</strong>
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<div>Researchers at the renowned Children’s Medical Research Institute (CMRI) in Sydney have identified a small molecule that can stimulate the function of the defective PGAP1 gene. "Initial lab tests are promising: It works", says Leszek Lisowski, head of the research Programme. "Now, the molecule is to be tested on disease models built using cells of affected children – a crucial next step towards a therapy." In fact, the last step required to initiate clinical studies.
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<div><strong>"We are very close and all kids affected by this ultrarare disease will be able to benefit from the results!"</strong>
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<div>To make this happen, we parents have built an international cooperation with the Department of Pediatric and Adolescent Medicine of the University Clinic Göttingen - UMG. “We see great potential in this new medical approach”, says Dr. Christin Johnsen, doctor at the UMG.
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<div><strong>Everything is ready – we only need a few more funds to finance these last steps on the way to a therapeutic breakthrough.</strong>
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<div><strong>Why We Depend on Private Support</strong>
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<div>Ultra-rare diseases are often “not profitable” for the pharmaceutical industry. That’s why there is no public funding, no support from healthcare companies.
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<div>Nevertheless, we have a very realistic chance to substantially improve the lives of our kids and all other affected kids with this breakthrough research, if not cure the disease at all.
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<div>Us parents have already invested a lot of our own money – but now we can’t do it alone anymore.
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<div><strong>Help Us Turn Hope Into Reality</strong>
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<div>We urgently need USD 100,000 to conduct the next tests and bring the therapy into clinical trial for our kids.
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<div>Every single cent goes directly to the research at CMRI in Sydney and the implementation of the therapeutic approach.
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<div><strong>How You Can Help – Every Donation Matters!</strong>
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<div>Whether it’s USD 10, 50, 100, or 500 – every contribution brings us closer to making a therapy possible for our children. Together, we can make it happen!
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<div>All gifts will support (gene) therapy research into PGAP1 led by Children’s Medical Research Institute, including ancillary costs – such as salary and salary on-costs, and research and project costs including laboratory materials, samples, consumables and equipment. 100% of the donations will be used for this critical research. <strong>No administrative fees </strong>are taken by Children’s Medical Research Institute.
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<div><strong>We’ve already come a long way – but we can’t move forward without you.</strong>
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<div><strong>Our Appeal to You</strong>
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<div>Help us give Emil, Mary, Jonathan, Charnice and Madison, as well as all other PGAP1 kids a real future.
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<div>A future with more possibilities, more inclusion – more life.
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<div><strong>Be part of this story. Donate now.</strong>
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<div><strong>watch our youtube explainer on PGAP1: </strong><a href="https://youtu.be/OMOYnh690VU" target="_blank" rel="noreferrer noopener"><a href="https://youtu.be/OMOYnh690VU" target="_blank" class="" rel="nofollow noopener noreferrer">https://youtu.be/OMOYnh690VU</a></a>
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