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Mr. PAUL. My nephew Mark Pyeatt has neurofibromatosis 2, NF2, but that is not who he is. He is an indomitable spirit, a courageous young man, a man who knows and faces each day certain that he is one with his God. He is like many young people on Earth--he is in search of the truth. He reads, he thinks, but he no longer hears.
Neurofibromatosis 2 is characterized by recurrent neurologic tumors. Its signature tumor affects the auditory nerves and destroys the hearing. Its relentless course eventually takes all of the hearing. I have never heard Mark complain.
While my signing is only rudimentary, most of his immediate family are proficient, and at Christmas dinner for 40 family members, nearly everyone is trying to learn some signing. The grandkids sing, ``Happy Birthday, Jesus. I am so glad you came.'' The whole family is learning to communicate with their hands. I mostly like to learn insults so I can taunt Mark on the golf course. I can't use most of the signs he taught me on the Senate floor. I don't know this for certain, but I think the seven words George Carlin said you can't say on TV, I think you can't sign them on TV either. I love the way names for people in sign language are created only by the deaf. Mark's mother Lori is ``L'' to the ear because she is on the phone all the time. My wife Kelley is ``K'' sweet. My middle son Duncan is ``D'' in a hoop because he likes basketball.
Neurofibromatosis 2 is a rare disease. Some call it an orphan disease. Orphan diseases face certain obstacles that others do not. Money is typically allocated to research based on how prevalent a disease is. For rare diseases, the resources are likewise rare.
In order for investors to invest in a cure for neurofibromatosis 2, regulatory obstacles need to be cleared. We need to allow foreign drug studies to be accepted in the United States and not repeated. We need to have speedy approval of drugs that are already being used by the general population in other countries.
My chief of staff's sister Karen has pulmonary fibrosis--another orphan disease.
She is 40 years old with a young daughter, and she is likely only alive today through a fluke in the system. She takes a medication that is part of an experimental trial in the United States but has been on the general market in Japan for years. If she didn't live near a research center and if her family couldn't afford to pay $1,500 a month out-of-pocket, she wouldn't receive this drug, even though it is legal in Japan.
The drug should have been cleared already, but we are not doing a good enough job of trying to get drugs cleared. It went through trials here. It has already been approved in Europe and Japan, but 200,000 Americans who have a rare deadly terminal disease are being denied this drug.
We all want safety in the drugs and in the cures for disease. We all acknowledge this is a balancing act. We should all acknowledge the regulatory obstacles and burdens new drugs face in our country are oppressive and counterproductive.
My hope is by putting a face to two orphan diseases--my nephew Mark, with neurofibromatosis, and my staff member's sister Karen, with pulmonary fibrosis--this situation will be made more personal. These are people who are close to our families, and we hope others will come to realize we must do something to get rid of government obstacles to cures for rare diseases.
I yield the floor.
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