Experts say lives are being put at risk by a “lottery” for people with the rare blood disorder paroxysmal nocturnal haemoglobinuria (PNH), which affects around 12 patients north of the Border.
The disorder causes red blood cells to explode, leaves patients too exhausted for simple tasks and at risk of kidney failure and potentially fatal blood clots. Median survival from diagnosis is 10 to 22 years. In at least two cases, NHS Greater Glasgow and Clyde (NHS GGC) has refused to fund the first treatment, called eculizumab (a monoclonal antibody), ever approved for patients with the condition.
