Meeting the ambitions of the UK’s Rare Diseases Framework: Insights from the Gold Standard National Paroxysmal Nocturnal Haemoglobinuria (PNH) Service
Paroxysmal Nocturnal Haemoglobinuria (PNH) is a chronic, rare, acquired blood disorder characterised by the susceptibility of blood cells to attacks from the body's immune system.1 The life-threatening condition involves the destruction of red blood cells which can lead to various symptoms, including fatigue, anaemia and blood clots.2 It is estimated that there are between 650 to 900 people in England living with the condition.3
This report, informed by the insights of experts from the National PNH Service, looks into PNH care in the UK, evaluating the current care and treatment landscape for PNH against the ambitions detailed in the Government’s Rare Diseases Framework and Action Plans. The report considers where there have been significant successes and where lessons can be learnt for other rare conditions.
Read the full report here.