Trisha is the only known child in Australia with an ultra-rare, fatal disease -Aspartylglucosaminuria (AGU). She is one of 120 known cases across the world, who desperately need a clinical trial to find a cure.
The medicine in the clinical trial requires funding and it comes with a heavy cost - a price tag of US$3.5 million. From Australia, we pledge to raise $1 million.
Due to the complexity, extreme rareness and limited data on AGU, it's almost impossible to attract the necessary interest and funding from big investors/medical research organisations.
To give Trisha a fighting chance, we're taking matters into our own hands alongside other AGU families to fund the trial ourselves. We're asking you to join the fight with us; we need your help to find a cure for our daughter and few AGU kids across the globe.
The difficulty with this genetic neuro-degenerative disease is that symptoms don't begin to show until two or three years of age and that's what happened Trisha. She was a healthy baby girl until she was 15-months-old and could barely walk and then we discovered her learning, physical ability and development were falling behind too.
Trisha has never been able to play outdoor games with her friends; she requires support with basic daily activities and struggles to keep up in the classroom. This doesn't stop her from having a love for swimming, arts and cooking - she's a real foodie!
Since we found out about Trisha’s disorder, our life has never been the same. Trisha is a very lively girl who enjoys dancing and singing, however it's heartbreaking to see her not being able to read, write and play like her younger sister. We want to give her the opportunity to thrive.
We can't do this alone. We need your help to fund the clinical trial to cure AGU for Trisha and children across the world. Please share, donate and support Trisha's journey - every little bit helps #SaveTrisha
You can read more about Trisha in the media articles covered in a few popular Australian news websites:
How your donations will be spent:
US$3.5 million is needed to produce the necessary medicine for the clinical trial to be carried out at a University Hospital in the United States. This will be able to treat children who participate in trials 1 and 2.
• April 2021 - Lock production spot [secure a spot in a manufacturing facility to produce medicine for AGU clinical trials.]
• August 2021 - Toxicology [The primary objective of toxicology studies in the drug development process is to evaluate the safety of potential drug candidates. This is accomplished using relevant animal models and validated procedures.]
• Jan 2022 - IND application FDA approval [Investigational New Drug Application (IND) And Food and Drug Administration (FDA)]
• April/May 2022 - Patient 1 starts clinical Trial
• June/July 2022 - Patient 2 starts clinical Trial
The goal of this GoFundMe is to collectively help raise the $3.5 million with other fundraising efforts. We are making joint efforts with eight families around the globe, including GoFundMe campaigns in the USA, Canada, Switzerland, Spain, and France
Additional funds collection will be needed later to pay for hospital expenses during the clinical trials. Which could be an additional US$2million.