Friedreich Ataxia
Friedreich Ataxia (FA) is a progressive neurological disease with symptoms commencing in children as early as 1 years old. The genetic condition causes deterioration in coordination, walking, speech, hearing and vision. After several years, children are often unable able to walk and they lose fine motor skills. Many children also develop diabetes, scoliosis and ultimately life threatening heart conditions. There is currently no treatment available in Australia, or cure.
Co-founders of TARFfa, Mason and Natalie Allamby are parents of three young children. One of their children, Tristan, has Friedreich Ataxia. Tristan is one of the youngest people in the world to be diagnosed - just after his 4th birthday.
Our Work
Working with the best scientists and neurologists around the world, TARFfa funds research. TARFfa collaborates with charities focused on treating and curing Friedreich Ataxia.
Funding scientific research and trials is often through government grants or generous donations by families impacted by Friedreich Ataxia. This is often a long and arduous process, one that contributes to the length of time it takes for research to become a treatment. This is where TARFfa will speed up the process of research and trials, as the funding is already available.
TARFfa receives no government funding.
Our Latest Trial
TARFfa funding of a clinically approved medication - Dumethyl fumerate (DMF) is on track to become the first treatment already in application for other conditions. This will reduce the approval time to become a treatment for FA, and in some circumstances will result in the medication being given immediately to patients on compassionate grounds.
Forty patients are currently being recruited for a double-blind, randomized, placebo-controlled 3 month trial, located in Italy, Principal Investigator, Professor Sacca and his team have previously shown that patients DMF can:
- Protect FA patient cells.
- Rescue mitochondrial biogenesis deficiency.
- Increase frataxin expression through improving gene function.
This Phase 2 trial, funded by TARFfa, will test the efficacy, safety and tolerability of Dimethyl Fumarate in Friedreich Ataxia (DMF-FA-201). The trial is in collaboration with the institution: Universita delgi Studi di Napoli “Frederico”, Dipartimento di Neuroscience,
Scienze Riproduttive e Odontostomatologiche, Napoli, Italy, and FARA.
Contact Us
We would like to hear from you. Please contact Co-Founder and CEO Natalie Allamby.