Currently, there are 15 listed treatments available for 10 different rare diseases which are funded via the Life Saving Drugs Program.
In order for a patient to qualify for funded treatment, they must meet a certain criteria and apply with support of their treating doctor.
Agalsidase alfa (Replagal®), Agalsidase beta (Fabrazyme®) and Migalastat (Galafold®) for the treatment of Fabry Disease
Laronidase (Aldurazyme®) for the treatment of Mucopolysaccharidosis Type I (MPS I)
Idursulfase (Elaprase®) for the treatment of Mucopolysaccharidosis Type II (MPS II)
Elosulfase alfa (Vimizim®) for the treatment of Mucopolysaccharidosis Type IVA (MPSIVA)
Galsulfase (Naglazyme®) for the treatment of Mucopolysaccharidosis Type VI (MPS VI)
Alglucosidase alfa (Myozyme®) for the treatment of Infantile-onset, Juvenile Late-onset or Adult Late-onset Pompe Disease
Eculizumab (Soliris®) for the treatment of Paroxysmal Nocturnal Haemoglobinuria (PNH)
Nitisinone (Orfadin®) for the treatment of Hereditary Tyrosinaemia Type I
Brineura® (Cerliponase Alfa®) for the treatment of Batten Disease
Rare Voices Australia (RVA) is a national, not-for-profit organisation established in 2012 with a vision to be ‘the unified voice for ALL Australians living with a rare disease’.
RVA is Australia’s national alliance advocating for those who live with a rare disease.
RVA provides a strong common voice to promote health policy and a healthcare system that works for those with rare diseases.