Porphyria Treatment Market Dynamics
The most common tests used for porphyrias diagnosis are measurements of substances such as porphyrin precursors and porphyrins, in blood plasma, red blood cells, feces, and urine. Measuring enzymes in cells and mutations in DNA is useful for confirmation and family studies. Though, no cure is available for porphyria, there is treatment for each type of the disease. Treatment depends on the type and severity of the disorder and can involve treating with heme, giving medicines to relieve the symptoms, or drawing blood. Drugs like Thorazine, Chlorpromazine, Panhematin and Hemin can be used to treat symptoms of porphyria. Panhematin (Recordati Rare Diseases) is used for treating acute porphyrias. It is a lyophilized form of alkaline heme that has to be reconstructed immediately before administration and should be infused into a large peripheral vein. As per American Porphyria Foundation, Panhematin is the only commercially available heme therapy in the U.S.
Porphyria Cutanea Tarda (PCT) is the most common and treatable form of porphyria and occurs, due to deficiency of the enzyme, uroporphyrinogen decarboxylase (UROD). PCT is a genetic disorder, however, some people have a genetic (autosomal dominant) deficiency of UROD that results in growth of PCT. Most people with the inherited enzyme deficiency remain latent and show no symptoms. Various environmental factors can impact the occurrence and severity of symptoms of porphyria. Smoking, alcohol, change in hormones levels, stress, certain drugs, and dieting or fasting can trigger the signs and symptoms of some forms of porphyrias. Moreover, exposure to sunlight severely damages the skin of people with cutaneous porphyrias. However, low incidence rates of porphyria can negatively impact the porphyria treatment market
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