Generic drugs to treat four rare diseases launched

  • November 25, 2023
  • Posted by: OptimizeIAS Team
  • Category: DPN Topics
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Generic drugs to treat four rare diseases launched

Subject : Science and Tech

Section: Health

Context:

  • The Union Health Ministry has made available generic drugs to support the care and treatment of four rare disease: Tyrosinemia-Type 1, Gauchers Disease, Wilson’s Disease, and the Dravet-Lennox Gastaut Syndrome.
  • The cost of these drugs will be slashed by anywhere between 60 and 100 times of their current market value.

Details:

  • The ministry is also considering release of generic drugs for two more rare diseases: Phenylketonuria and Hyperammonemia.
  • Focus is on sickle cell disease, and on the syrup for children under five who can’t be administered tablets.

Rare Disease:

  • A rare disease is a health condition of particularly low prevalence that affects a small number of people.
  • It collectively afflicts 6-8% of the population in any country at any given time, so India could have 8.4 crore to 10 crore such cases.
  • Nearly 80% percent of these diseases are genetic in nature.
Rare DiseaseDescription
1. Tyrosinemia-Type 1It is a rare autosomal recessive genetic metabolic disorder characterized by lack of the enzyme fumarylacetoacetate hydrolase (FAH), which is needed for the final break down of the amino acid tyrosine.
2. Gauchers DiseaseIt is the result of a buildup of certain fatty substances in certain organs, particularly your spleen and liver. This causes these organs to enlarge and can affect their function. The fatty substances also can build up in bone tissue, weakening the bone and increasing the risk of fractures.
3. Wilson’s DiseaseIt is a rare, autosomal recessive disorder caused by abnormal copper accumulation in the body particularly involving the brain, liver, and cornea.
4. Dravet-Lennox Gastaut SyndromeIt is a genetic epilepsy.
5. PhenylketonuriaIt is a rare inherited disorder that causes an amino acid called phenylalanine to build up in the body.
6. HyperammonemiaIt is a metabolic condition characterized by the raised levels of ammonia, a nitrogen-containing compound.

Source: The Hindu

Generic drugs to treat four rare diseases launched Science and tech