Finally Rare and Neglected Diseases will be Addressed

Money drives research.Ai?? Cellular phones, for example, have advanced to the point where they can do nearly everything that a computer can do. Despite the advances made in cell phone technology we still lack efficient and affordable solar energy technology.Ai?? Companies are very willing to fund research into cell phone advancement because market demands mean that any money spent on research and development will be quickly recuperated as profit.Ai?? If the same market existed for solar panels as exists for cell phones perhaps companies would spend the research dollars to develop solar technology.


The same logic applies to medicine.Ai?? Drug companies focus primarily on research that will generate the largest profit; therefore the push is to develop drugs that treat common diseases and afflictions.Ai?? There are almost 7,000 recognized human diseases but only about 300 receive attention from pharmaceutical research firms.Ai?? The other 6,700 diseases are considered unprofitable and are largely ignored by the big drug companies.Ai?? About 6,000 of those diseases are considered rare and the rest are considered neglected.Ai?? A rare disease is one defined by the Orphan Drug Act as affecting less than 200,000 people in the United States.

Neglected diseases are those that typically affect poorer people or other often ignored subsets of the population.Ai?? Neglected diseases typically spread in areas with substandard housing, unsafe drinking water, poor sanitation, and limited access to health care. These diseases are common in low-income nations in Africa and Latin America and are also seen in areas of extreme poverty in the United States.


The United States Government has established the Therapeutics for Rare and Neglected Diseases (TRND) program through The National Institutes of Health (NIH) to address the paucity of research into treatment for these rare and neglected diseases.Ai?? The TRND was funded with $24 million for fiscal year 2009. Ai??A goal of the program is to initiate and facilitate the development of drug therapies for rare and neglected diseases.

Study Focus

Some of the diseases currently being researched are:

  • Niemann-Pick C
  • Hereditary inclusion body myopathy
  • Sickle cell disease
  • Chronic lymphocytic leukemia
  • Duchenne muscular dystrophy
  • Fragile X syndrome
  • Cryptococcal meningitis
  • Core binding factor leukemia
  • Neonatal herpes simplex
  • Autoimmune pulmonary alveolar proteinosis
  • Fibrodysplasiaossificans progressive
  • Schistosomiasis
  • Creatine transporter defect

For more information see http://www.ncats.nih.gov/research/rare-diseases/trnd/trnd.html

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