Government's multi-million dollar announcement

Patients living with ultra-rare condition, Morquio A Syndrome, will soon have free access to life-saving drug Vimizim thanks to the Government's recent multi-million dollar announcement.

Over the next five years, the Australian Government will be investing $44 million to list the drug on the Life Saving Drugs Program, helping to significantly increase life expectancy for those living with the disease.

Morquio A Syndrome is only known to affect 21 Australians and just 3000 people worldwide. The life-threatening condition occurs when the body is missing or does not have enough of a vital enzyme needed to breakdown materials in the body. As a result, the materials eventually build up in tissues, bones, and major organ systems, causing potentially serious problems, including heart disease, skeletal abnormalities, vision and hearing loss, difficulty breathing, and early death.

“Due to the progressive nature of Morquio A syndrome, early diagnosis may be critical to optimise patient outcomes, and Vimizim is the only available treatment option for this ultra-rare disease,” Associate Professor Carolyn Ellaway, Paediatric Specialist at the Genetic Metabolic Disorders Service of The Children's Hospital at Westmead and a Vimizim clinical trial investigator, said.

“The listing of Vimizim on the Life Saving Drugs Program offers Australians living with Morquio A the potential for better endurance as demonstrated in the clinical trials,” Associate Professor Ellaway said.

The drug will be available as part of the program from August 1.