Alone We Are Rare, but Together We Are Strong: Orphan Diseases Quiz

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About This Quiz

They affect between 420 and 560 million people around the world, yet account for less than 10 percent of global pharmaceutical spending. Take this quiz to see how much you know about conditions known as "orphan" diseases and how they're treated.

Is it true that rare, or "orphan," diseases are typically those the pharmaceutical industry won't profit from?

True. Some common diseases might be overlooked because they're considered orphan diseases, but often these diseases are ignored because they aren't a public health need.

How many people does a disease affect to be considered orphan?

According to the FDA, an orphan disease is one that affects fewer than 200,000 Americans.

More than 25 million Americans are affected by how many rare diseases?

There are 8,000 known orphan diseases affecting people in the U.S.

True or false: Out of about 7,000 diseases, only about 400 have an effective drug therapy.

Of the 7,000 or so rare diseases affecting Americans, drug therapy is available for about 400.

True or false: The plot of two "Quincy, M.E." episodes focused on rare disease awareness.

Both "Seldom Silent, Never Heard" (1981) and "Give Me Your Weak" (1982) focused on rare and orphan diseases.

True or false: Actor Jack Klugman testified before Congress in an effort to draw attention to orphan diseases and treatments.

Yes, it's true. Reagan went on to sign the Orphan Drug Act of 1983.

What does the Orphan Drug Act of 1983 provide to pharmaceutical companies?

It provides incentives for the pharmaceutical industry to invest in developing treatments for rare diseases.

Which of the following is NOT considered an orphan disease?

All of these are considered orphan diseases.

What condition may cause frequent lung infections, an inability to sweat and "reverse freckles"?

XLPDR, an extremely rare skin disease, is known to affect 14 families worldwide.

Which disease, which affects about 1 in 2 million people, is sometimes called stone man disease?

Fibrodysplasia ossificans progressiva (FOP), a rare connective tissue condition, is sometimes called stone man syndrome.

Which rare condition causes a person to begin rapidly aging as a child?

The appearance of accelerated aging as a kid happens with the genetic condition Hutchinson-Gilford Progeria Syndrome.

How are most orphan diseases acquired?

Most of these rare diseases are inherited. The remaining are associated with environmental conditions.

How is an orphan disease defined under the Orphan Drug Act?

They're diseases for which "there is no reasonable expectation that the cost of developing and making available in the U.S. a drug for such disease or condition will [be] recovered from sales in the United States of such drug."

What is the characteristic symptom of the condition called Jumping Frenchmen of Maine?

The startle response can consist of jumping, screaming and throwing objects.

What is the name of the disorder that causes blue skin and chocolate-colored blood?

Methemoglobinemia, most famously seen in Appalachia's Fugate family, causes blue skin.

How many orphan drugs have been developed as a result of the Orphan Drug Act of 1983?

More than 250 new drugs to treat these diseases have been developed since the act went into effect.

What rare neurodegenerative disease did the 2014 Ice Bucket Challenge benefit?

2014's Ice Bucket Challenge raised more than $100 million for ALS research.

Which adrenal condition may cause your skin to appear tan?

People with Addison's disease may develop a dark skin tan.

What percent of breast cancer diagnoses are male breast cancer?

Less than 1 percent of all breast cancer diagnoses are in men, and less than one percent of male cancers are breast cancers.

Which rare childhood neurodevelopmental disorder caught the world's attention when it was linked to the Zika virus in 2016?

Awareness of microcephaly, a rare disorder causing a significantly smaller-than-average head circumference, grew in 2016 due to the Zika virus.

Which fatal neurological disease are you probably safe from unless you eat human brains?

To get kuru, or "laughing disease," you'd have to eat the contaminated brain of a remaining carrier living in the remote highlands of New Guinea.

Which rare mental illness is sensationally known as "walking corpse syndrome"?

Cotard's delusion is a mental illness causing people to believe they are dead (or somehow don't exist).

True or false: You can literally be allergic to the cold.

Contact with cold air or water causes cold urticaria, allergy symptoms similar to those with bees or peanuts.

Which condition, perhaps the most rare disease of them all, is named after the twins diagnosed with it?

The progressive muscle disorder, Fields Condition, affects Kirstie and Catherine Fields.

True or false: It's possible for a condition initially classified as "orphan" to outgrow that classification.

For instance, HIV/AIDS emerged as a rare disease.

True or false: It's possible for a common condition to be reclassified as rare.

With vaccination and other preventative methods, some common conditions may be reversed and become rare.

What does NORD stand for?

NORD, the national advocacy group for rare diseases, stands for National Organization for Rare Disorders.

Which of the following incentives is available for orphan drug development?

All of these options are available for drug development.

Devices intended for diagnosing or treating a condition that affects fewer than 4,000 Americans are called what?

These are known as Humanitarian Use Devices (HUD).

When is Rare Disease Day observed?

Since 2008 Rare Disease Day has been celebrated on the last day of February.

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