Rare diseases are Common
I had the privilege of interviewing two public health advocates in my Manhattan office recently, Mary Dunkle and Denis Costello. These two enthusiastic and dedicated educators brought me up to speed on an issue I knew little about: rare diseases.
A rare disease is defined as one that afflicts fewer than 200, 000 people in the U.S. Often, physicians and pharmaceutical companies are unable to deal adequately with rare diseases as they are so little understood.
There are now over 7, 000 diseases listed as "rare." These represent all types of diseases, including many forms of cancer. A rare disease can have a population as few as one person.
I quickly learned that rare diseases have become so common as a class that there is now a collective effort to assist those afflicted and their families, and to educate the public on their complexities. Unbelievably, they are so common that one in ten Americans is afflicted today by a rare disease!
In the U.S., that works out to about 30 million Americans. In Europe, the number is almost identical. The issue has become so large that the end of February has been designed globally as "Rare Disease Day."
Rare diseases are sometimes associated with celebrities such as Lou Gehrig, who died of a disease known as ASL, or John Travolta, whose teenage son recently died of a rare disease. Others are depicted on TV, like the one that prematurely ages children. Another afflicts children with Alzheimer's, ordinarily the disease of the very old.
Rare diseases are not rare to the patients they afflict. As one patient reported, "I was born without being able to breathe, so the hospital staff had to put in a breathing tube. I was also born without being able to suck or move my arms or legs. I had to learn every basic motor function through hours of physical therapy. I was born with Dandy-Walker Syndrome and hydrocephalus."
Dandy-Walker Syndrome, one of 7, 000 identified rare diseases, is a neurological condition. Individuals with this syndrome are born with an underdeveloped cerebellum and the body's natural way of draining spinal fluid off the brain is obstructed causing hydrocephalus - excess fluid on the brain.
In 1983, Congress passed the Orphan Drug Act, providing incentives to pharmaceutical companies to develop medications for rare diseases such as Dandy-Walker Syndrome. During the 10 years prior to this Act, there were, shockingly, only 10 products developed for these many "orphaned" diseases. Today, because of the Act, there are over 1, 700 in the research pipeline and 330 have been approved. Senator Ted Kennedy was an early advocate for "orphan" drug legislation.
A second Act, the Rare Diseases Act of 2002 provided federal funding for research. Also, the U.S. Food and Drug Administration (FDA) now provides grants for "orphan" drug product development.
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What is an example of a disease that attacks the human immune system
There are many diseases that destroy the immune system. The most widely-spread immune disease is the AIDS virus affecting huge populations in third world countries especially in the continent of Africa.