I’m 35 and my best friend, whose birthday is the same as mine, has been diagnosed with a rare disease call PNH. Are you able to explain what it actually is and if there’s a cure at this stage?
Dani Jonsson, via email.

APNH stands for paroxysmal nocturnal haemoglobinuria. It’s quite a rare disease and very serious. PNH causes the blood cells to break down, which leads to disabling fatigue, clots forming in arteries, anaemia and chronic kidney failure. The disorder is also associated with other conditions under the leukaemia umbrella.

The good news for the around 200 Aussies affected by PNH to varying degrees is that a new medication, Soliris, has recently been approved for use.

The condition used to be treated with regular blood transfusions and medicine to slow down the immune system. But it’s hoped this new drug will cut down clots, reduce fatigue and decrease the need for patients to have transfusions.

Although it’s yet to be fully proven, this medication may extend the lives of people suffering from debilitating PNH, and that’s a wonderful prospect.