Rare Voices Australia (RVA) attended the launch of a world-first clinical trial for people living with Ataxia-Telangiectasia (A-T) at Wesley Medical Research in Brisbane with the Hon. Greg Hunt MP, Minister for Health and Aged Care; the clinical trial team led by Dr David Coman and Prof Martin Lavin; RVA Partner BrAshA-T; and children living with A-T and their families.
RVA is pleased to partner in this research as a member of the Trial Steering Committee. This trial is funded through the Medical Research Future Fund (MRFF). Previously, RVA’s advocacy influenced the creation of the MRFF Rare Cancers, Rare Diseases and Unmet Need Clinical Trials initiative.
As there are currently no effective treatments to treat A-T, this trial is an important milestone for the A-T community. As highlighted in the National Strategic Action Plan for Rare Diseases, participation in a clinical trial is often the only way to access treatment for many people living with a rare disease.
What is A-T?
A-T is a rare genetic metabolic disorder with an incidence of 3 in 1 million births. A-T is a life-threatening disorder that causes cerebral palsy, muscular dystrophy, and immune deficiency leading to a cystic fibrosis-like lung disease and cancer. A-T progresses rapidly, robbing children of their ability to walk, speak and swallow—their minds remain unaffected. There are currently no effective therapies to treat A-T.
For more information about A-T, support or to learn more about this clinical trial, please visit BrAshA-T’s website.