EBoard Members
Who are the freshman EBoard members?
Amir, Keerthana, Audrey, Sonia
What is an orphan drug?
a rare disease that only has one available treatment on the market
When was Rare Disease Day launched?
2008
How many years on average does it take for a rare disease patient to get diagnosed?
6 -8 years
Which famous swimmer has marfan syndrome?
Michael Phelps
List off Amir's favorite hobby that he presented.
Rare disease advocacy, lifting, and running
What portion of all new drugs approved by the FDA are for the treatment of rare diseases?
1/3
What is the name of the 1983 US law passed to increase incentives for rare disease treatment development?
Orphan Drug Act
All pediatric cancers are classified as rare diseases true or false?
True
What rare disease does Selena Gomez have?
Lupus
How many schools did Audrey transfer to in one school year?
4
How many drugs for rare diseases were approved by the FDA before the Orphan Drug Act of 1983?
A. 12
B. 27
C. 38
D. 67
David Fajgenbaum, MD, MBA
What percentage of rare diseases are genetic?
80%
This 5x Wimbledon and 2x US open winner in singles women's tennis has Sjogren's Syndrome?
Serena Williams
Which NBA player's son has Aaron played against in high school?
Lebron James
What percent of rare diseases have no FDA approved drug treatment?
95%
In his 1965 paper describing what is now called Angelman syndrome, pediatrician Harry Angelman said he was inspired to publish after seeing a Renaissance painting in Verona. Which painting was it?
Giovanni Francesco Caroto’s A Boy with a Puppet
Which cuddly name is given to a very non-cuddly rare disease?
PANDAS
What rare disease does the main character August in the book/movie Wonder have?
Treacher Collins Syndrome
Which EBoard member is adopted from China?
Cayla
What is Pompe disease treated with?
A. Stem cell therapy
B. Immunoglobulin therapy
3. Enzyme Replacement Therapy
4. Subcutaneous Therapies
What is the first recognized rare disease?
Alkaptonuria (1902)
This ancient clinical sign, describing sweet smelling urine, helped inspire the name of a rare metabolic disorder?
Maple Syrup Urine Disease
What rare disease does Lady Gaga have? It's a chronic condition characterized by intense "fibro fog".
Fribromyalgia