India reports far fewer people with orphan disease

Subject: Science and tech

Section: Health

Rare disease or Orphan disease:

A rare disease is a disease that affects a small percentage of the population.
In some parts of the world, an orphan disease is a rare disease whose rarity means there is a lack of a market large enough to gain support and resources for discovering treatments for it, except by the government granting economically advantageous conditions to creating and selling such treatments. Orphan drugs are ones so created or sold.
Most rare diseases are genetic in origin and thus are present throughout the person’s entire life, even if symptoms do not immediately appear.
Many rare diseases appear early in life, and about 30% of children with rare diseases will die before reaching their fifth birthdays.
No single number has been agreed upon for which a disease is considered rare.
Global Genes has estimated that currently approximately 10,000 rare diseases exist globally, with 80% of these having identified genetic origins.

Initiatives by patient groups:

Hospitals in India have so far reported less than 500 of these diseases.
The Government’s National Policy for Treatment of Rare Diseases has only recently started making its mark.
Diseases prevailing in our countries include cystic fibrosis, hemophilia, lysosomal storage disorders, sickle-cell anemia, etc.
DART, the Dystrophy Annihilation Research Trust, a body formed by parents of patients suffering from Duchenne’s muscular dystrophy. In this condition, muscles in the pelvis begin to waste away from the age of three.
In partnership with the IIT and AIIMS located in Jodhpur, the Trust has begun a clinical trial of an efficient and personalized antisense oligonucleotide-based therapeutic regimen for this dystrophy.

Leprosy free India:

leprosy is now considered a rare disease in India.
Incident rate: 0.45/10,000 population.
Recent research on the synthetic antibiotic rifapentine, which is widely used against tuberculosis, has shown that a single dose of this drug, when administered to household relatives of a leprosy patient, significantly curtailed the spread of leprosy to them over a four-year study period
India envisages to eradicate leprosy by 2027.