Rare Disease Day and Biotech


28-Feb-2018 22:26 PM



     February 28, 2018 is Rare Disease Day. What is Rare Disease Day? The main objective of Rare Disease Day is to raise awareness among the general public and decision-makers about the rare diseases and their impact on patients' lives. Definition off from

How rare is rare disease?

  • A disease or disorder is defined as rare in Europe when it affects fewer than 1 in 2,000.
  • A disease or disorder is defined as rare in the USA when it affects fewer than 200,000 Americans at any given time.
  • 80% of rare diseases have identified genetic origins whist others are the result of infections (bacterial or viral), allergies and environment causes, or are degenerative and proliferative
  • 50% of rare diseases affect children
  • 30% of children with rare disease will not live to see their 5th  birthday
  • Rare diseases are responsible for 35% deaths in the first year of life
  • There are approximately 7,000 different types of rare diseases
  • 30 million people in the USA are living with rare disease

Where are the cure?

  • In 2017, there are 476  Orphan Drug Designations
  • Orphan Drug Act of 1983 is law passed in the U.S.. to facilitate development of orphan drugs
  • There are 46 Novel Drug Approval in 2017. List of FDA 2017  Orphan Drug Approval

Ten Publicly Traded Rare Disease Stocks: (Not a complete list)

  • Amicus Therapeutics Inc (FOLD:NASDAQ)
  • Sarepta Therapeutics Inc (SRPT:NASDAQ)
  • Incyte Corp (INCY:NASDAQ)
  • Insmed Inc (INSM:NASDAQ)
  • Ultragenyx Pharmaceuticals Inc (RARE:NASDAQ)
  • Acceleron Pharma Inc (XLRN:NASDAQ)
  • Novelion Therapeutics Inc (NVLN:NASDAQ)
  • MyoKardia Inc (MYOK:NASDAQ)
  • aTyr Pharma Inc (LIFE:NASDAQ)
  • Abeona Therapeutics Inc (ABEO:NASDAQ)

List of all rare disease

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