Currently there are 12 listed treatments available to 8 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.
Imiglucerase (Cerezyme®), Velaglucerase (VPRIV®), Taliglucerase (Elelyso®) and Miglustat (Zavesca®) for the treatment of
Gaucher disease (Type 1)
Agalsidase alfa (Replagal®) and Agalsidase beta (Fabrazyme®) 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)
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 (HTI)
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.