The Australian Paroxysmal Nocturnal Hemoglobinuria Therapeutics Market size stood at around USD XX billion in 2020 and is projected to reach USD xx billion by 2028, exhibiting a CAGR of xx% during the forecast period.
Paroxysmal Nocturnal Haemoglobinuria (PNH) is a very rare and debilitating disease of the bone marrow that affects the blood and major organs. It is a chronic, life-threatening illness and can have devastating effects upon a patient physically, mentally and emotionally.
PNH is an acquired illness caused by an injury to a bone marrow stem cell and is not hereditary. The bone marrow produces all blood cells for the body, being white cells, red cells and platelets. PNH occurs when a gene required for producing normal blood cells becomes mutated resulting in a PNH clone of bone marrow cells that produce defective blood cells. The white blood cells and platelets do not function as effectively as normal cells, leaving patients prone to infections and blood clots. While all three types of blood cells are affected by PNH, most of the symptoms experienced by patients are due to the destruction of red blood cells.
PNH is believed to affect males and females in equal numbers, although some studies show a slight female preponderance. The prevalence is estimated to be between 0.5-1.5 per million people in the general population. The disorder has been described in many racial groups.
Factors driving the Paroxysmal Nocturnal Hemoglobinuria Therapeutics market include rise in number of blood & bone marrow related disorders, increase in aging population, growing disposable income coupled with unmet need of treatment of PNH and technological advancements in stem cells transplantation. In addition to this, the emergence of biologics and adoption of novel therapeutics are further pushing the market growth. However, increase in cost of medical equipment, specifically surgical equipment required for stem cell transformation; lack of reimbursement policies; and occurrence of side effects in related current available treatments may hamper the Paroxysmal Nocturnal Hemoglobinuria Therapeutics market.