PNH Support Association of Australia (PNHSSA) says, a group of Australians living with an extremely rare disease are in an unfortunate place due to the recent government's decision not to fund for its treatments.

Paroxysmal Nocturnal Haemoglobinuria (PNH), a life-threatening disorder - that affects a group of 70 Australians - is characterized with red blood cells destruction that triggers a range of major health issues like blood clot formation, raised blood pressure, damage or failure of organs.

The only treatment for PNH is through the use of monoclonal antibody, soliris or eculizumab that protects blood cells from immune destruction.

One in three people with PNH will die within five years after diagnosis without treatment, said Professor Jeffrey Szer, director of the Department of Clinical Haematology and Bone Marrow Transplant Service at the Royal Melbourne Hospital.

Prof Szer says, If PNH struck Australians at the same rate diabetes or heart disease do, the government would not hesitate to open its wallet and fund the necessary medical care.

He stated there's no reason for people affected with a rare disease to be left without hope, just because only a limited number of them have the condition.

Medicine