Rare Disease Day and Biotech
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 Rarediseaseday.org https://www.rarediseaseday.org/article/what-is-rare-disease-day
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 https://globalgenes.org/raredaily/168445-2/
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 https://globalgenes.org/rarelist/
Source for this blog:
- Rare Disease Day http://www.rarediseaseday.org
- Rare Daily https://globalgenes.org/raredaily/
- Wikipedia https://en.wikipedia.org/wiki/Orphan_Drug_Act_of_1983