Seth and McKenzie Holder are brother and sister who have an inherited genetic terminal illness. This illness, Late Infantile Ceroid Lipofuscinosis, or Battens Disease, results in deterioration of the brain and eyesight, to the point where the child usually dies around the age of 10.
A clinical trial using Gene Transfer Therapy was held in New York and the Holder family were accepted to participate in the trial. This requires frequent trips and lengthy stays in New York for the children to undergo the treatment procedures in hope to battle the disease. The therapy requires manufacturing the gene they are missing, attaching it to a virus and implanting it into the brain in the hope it spreads and is able to manufacture the enzyme they need to stop the cells from dying.
WA Charity Direct assisted the family by funding the required follow up visits. The financial assistance allowed Seth, McKenzie and their parents to travel back to the US and to stay for the lengthy follow up treatments to the initial surgery.