While each rare disease only affects a small number of people itself, rare diseases as a whole are a serious threat to public health. Learn a little about some diseases that may not get big headlines, but present global problems.
What is considered a "rare" disease?
- a disease that affects fewer than 200,000 people
- a disease without a cure
- a disease that affects less than one in a million people
How many rare diseases are there?
What is another name for rare diseases?
- unusual illness
- orphan disease
- standalone sickness
When was the last time a rare disease was discovered?
- new, rare diseases are discovered every year
What are most rare diseases caused by?
- the environment
How many genes are in a human cell?
A third of people with rare diseases take how long to get a diagnosis?
- a year
- a year to five years
- They never get a correct diagnosis.
Why are rare diseases and drugs nicknamed "orphans?"
- Pharmaceutical companies abandoned funding for them, due to the relative low profit from such a small patient group.
- They were lonely.
- They had no genetic "parent" disease.
What did the 1983 Orphan Drug Act do?
- punished pharmaceutical companies
- guaranteed access to drugs
- created incentives and tax credits for research and manufacture of orphan drugs.
From 1973 to 1983, how many orphan drugs were developed with government assistance?
After 1983, how many drugs have been developed?
Let's jump into some rare diseases to discover more about them. Speaking of jumping, what is the name of one rare disease?
- Jumping Frenchman of Maine disease
- Leaping Lords disease
- Pouncing Cat disease
Some rare diseases are ones you actually might be familiar with. Which is NOT a rare disease?
- cystic fibrosis
- Lou Gehrig's disease
- Alzheimer's disease
While we think of cancer as a common disease, there are many forms of rare cancers. What is one?
Name the (real) rare disease.
- Hurting Hands Disorder
- Painful Legs and Moving Toes Syndrome
- Awkward Movement and Flinching Pain Disorder
What cuddly acronym is given to a very not-cuddly rare disease?
What is another rare disease with a provocative name?
- distant pancreas
- wandering spleen
- traveling throat
Let's see how good we are at medical terms. What is the clinical name when one of the blood vessels that supplies blood to the brain doesn't develop?
- Internal carotid agenesis
- neuro antihemo
- brain vessel gone
Some rare diseases affect disparate parts of the body. What is one disease that causes abnormalities in different regions?
- hand foot genital syndrome
- thumb liver craniofacial syndrome
- elbow knees and toes syndrome
Hantavirus is caused by what?
- a genetic mutation
- bad smog
- contact with rodent saliva, urine or droppings
What is one rare disease named, due to an abnormality that gives the iris a "keyhole" appearance?
- alien eye syndrome
- cat eye syndrome
- Margaret Keane syndrome
Parry Romberg syndrome causes what?
- autoimmune weakening
- small ears
- atrophy of the facial skin, tissue and muscles
Autosomal dominant hyper IgE syndrome (AD-HIES) causes blisters, rashes and scaling of the skin. What was it formerly called?
- snaking disorder
- Job syndrome
Kinky hair disease is now called Menkes disease, but it led to sparse, kinky hair. What caused it?
- a genetic condition that affects copper levels in the body
- too much protein
- not enough calcium
Proteus syndrome causes what?
- an ability to swim better
- tumor growth and bone and skin abnormalities
- webbed feet
Gigantism is caused by:
- an overactive pituitary gland
- overproduction of growth hormones
It has been speculated that Abraham Lincoln suffered from what rare disease?
- Marfan Syndrome
Cold urticaria causes what?
- an allergy to a sinus infection
- a growth that increases with contact with the cold
- an allergy to cold air or water
Rapid aging is seen in the rare disease:
- maturation abundance
- elderly growth syndrome
To raise awareness about rare diseases, the National Institutes of Health sponsors:
- Rare Disease Day
- a carnival
- a Formula One racing team