Rare Is Many
“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…
Expressing the CYP2U1 gene: A step closer to SPG56 Gene Therapy Success
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…
Our Researcher “Wolfie” secures $1M to test gene therapies for SPG56 using mini-brains
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…
A third patient sample for SPG56 research: Precious blood donations bolster hope for a cure
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…
Vale Emeritus Professor Alan Mackay-Sim
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…
How do we find a drug to treat SPG56?: High Throughput Screening: the next exciting step for SPG56 Drug Discovery
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….
Have we developed the cure?: SPG56 mini-brains dosed with gene therapy
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…
First scientific publication on GC4K’s research and SPG56 RNA sequencing underway
The first scientific publication about SPG56 stem cell research funded by Genetic Cures for Kids has gone live, published by our dedicated researchers Dr Hannah Leeson and Prof Ernst Wolvetang from Australian Institute for Bioengineering and Nanotechnology…
Hundreds of Queenslanders ran through Brisbane to rally awareness and funds to help us cure SPG56
What an amazing day! We are still reeling from the absolute joy of Saturday’s incredible event….so many amazing people and so many smiling faces at the event where three Brisbane…
