As this year draws to a close, we are holding a moment that once felt impossibly out of reach. It has been four solid years of research, collaboration, persistence and belief, and as many of our followers would know, last year we identified a gene therapy candidate for SPG56 that could be taken all the … Read more
We’re still glowing from the magic of our Shimmering Moon Gala—a night that truly lived up to its name. On a breathtaking full moon evening, 200 guests gathered to shine their light for a rare cause close to our hearts: curing SPG56 The energy in the room was electric. From the very first note to … Read more
Global Moonwalk unites 800 of ‘Tallulah’s Army’ under serendipitous full moon What an incredible journey we’ve been on together! On the Full Moon of 21 July, more than 800 amazing #TallulahsArmy supporters from around the globe came together for the Global Moonwalk for Our Moon’s Mission. From the USA, Nicaragua, Spain, Ireland and all across … Read more
“All the feels” after Rare Disease Day success in 2024 We are thrilled to share the amazing success of Rare Disease Day 2024 and whilst there’s a sea of #TallulahsArmy troops out there who we still don’t know personally, we want to give our heartfelt thanks to each of you who took part and made … Read more
Our Moon’s Mission has been dedicated to the pursuit of innovative gene therapy solutions for SPG56, the rare neurodegenerative disease that affects children like our young Tallulah Moon. Thanks to the hard work of top researchers worldwide, Genetic Cures for Kids has been making significant progress in developing a clean and effective viral vector for … Read more
We are absolutely over the moon that Our Moon’s Mission (OMM) organoid expert and lead researcher from UQ, Professor Ernst Wolvetang has secured almost $1 million from the Medical Research Future Fund (MRFF) to continue testing gene therapies with “mini-brain” organoids for children with SPG56! The monumental grant comes after Prof Wolvetang and his team … Read more
Thanks to the hard work of many, Genetic Cures for Kids’ researchers will soon recieve more blood samples from an SPG56 patient and her parents. From these precious samples, they will grow more organoids and help develop a cure for SPG56. When Tallulah was diagnosed as the only person in Australia with this ultra rare disease, … Read more
Chris, myself, our loving GC4K Board and Research Team at Our Moon’s Mission: Genetic Cures for Kids farewell the great and wonderful moustached man, Alan Mackay-Sim, with a deep sadness in our hearts. Alan passed away in early 2023, and lived until then, a life like no other, helping so many. Alan gave us a … Read more
The SPG56 drug discovery project has researchers working quickly to find an FDA approved drug that could be repurposed to treat SPG56 patients- possibly alleviating symptoms, or slowing/stopping disease progression. FDA approved drugs are preferred targets as they are easier to translate to clinical trial, and could also prove more accessible to the geographically dispersed … Read more
In more exciting research news for Genetic Cures for Kids (GC4K), the brain organoids that have been grown at AIBN from SPG56 patient stem cells have been dosed with the AAV9 viral gene therapy: the same one-dose therapy we are developing to treat patients with SPG56. At the end of September Dr Hannah Leeson received … Read more
