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The rare disease is that disease which affect a smaller number of people . The definition of rarefied disease is different in different countries and it qualifies on the groundwork of universe . It is potential that the rarefied disease in one country may not be a rarified disease in another country . particular Hiram Ulysses Grant are provided by the Government to companies who develop drug for rare diseases .
Is Myelofibrosis A Rare Disease?
Myelofibrosis is the condition in which the spongybone marrowtissue becomes sinewy resulting in the reduced production of various stemma cells . Myelofibrosis is a rarefied disease as it conforms to the criterion of the rare disease in many countries . Both primary myelofibrosis and secondary myelofibrosis are considered to be a rare disease .
junior-grade myelofibrosis is considered as rarified because the reason for secondary myelofibrosis i.e. polycythemia vera and essential thrombocythemia are themselves are rare condition . Almost 15 % of the patient suffering from polycythemia Vera develops myelofibrosis while only a few patients let of the essence thrombocythemia progresses to myelofibrosis . Myelofibrosis in the US has the preponderance of fewer than 200,000 patient and the relative incidence of the disease is approximately 3 patient per 100,000 populations.(1 )
Myelofibrosis Prognosis
The prognosis of this rare disease is unmanageable to value due to the dissimilar rate of progression and dissimilar symptom see by the patient role . Some multitude suffer from myelofibrosis do not have any characteristic symptoms and the disease is diagnosed through the quotidian ancestry test . Some patients have dangerous symptoms that affect their quality of life history . These patients experiencefatigue , depression , anxiousness , weaknessand recurring infection . Further , some patient respond favourably to handling while disease in some affected role fails to deal with the drug . The risk of human death is high in those patients whose disease progresses into acute myeloid leukemia , which is a extremely black shape . The prognosis of the patient depend upon various factors . geezerhood is one of the primary factor as young hoi polloi are able to recoup more quickly with the side effects of therapy and stem cell transplant is more successful in young people . More the type of chromosomal aberrancy more is the danger of patterned advance of the disease and poor the medical prognosis . Other factors affect prognosis include dependence on line transfusions , absolute frequency of grow infections and grade of platelets.(2 )
Rare Disease
The rare disease is the disease which affect a small universe at a given point in fourth dimension . Thus , not a very gamey number of people are affect by these disease . The uncommon disease is also known as an orphan disease in some jurisdictions . Due to a smaller number of patients useable for treatment , the government activity provides various subsidies for the companies doing research on drug for these disease . The drug for the orphan disease are know as orphan drugs . Most of the rare diseases are genetical diseases with ribose 5 - phosphate isomerase inadequacy is the rarest disease with only 3 patients diagnosed in the last three decades . The sound definition to qualify for a rarefied disease differs in unlike countries . In the USA , the disease which is present in less than 200,000 citizenry is term as a rare disease while in Japan ; the disease which dissemble less than 50,000 people is a rarefied disease . European Commission defines the rare disease in full term of low prevalence disease . Low preponderance is defined as the disease which affects 1 in 2000 people . Prevalence is generally used over incidence when defining the rare disease . The disease may be rare or common depending upon the area which is affected by the disease . For illustration , cystic fibrosis is a uncommon disease in India while it is a common disease in the European realm . Almost 80 % of a rare disease has genetics involved.(3 )
Conclusion
Both Primary and junior-grade myelofibrosis are chronic and rarefied bloodline disorders . The disease is less prevalent in the US and Europe . In the USA , the people dissemble by this disease are less than 200,000 . Prevalence of a disease is the criteria used for qualifying a uncommon disease .
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