07. Von Hippel-Lindau ( VHL ). VHL is said to affect one in every 35,000 people. It is a very rare genetic condition that is often characterized by the growth of numerous tumors in different parts of the body. It is an inherited disorder causing benign and malignant tumors to develop in the central nervous system and viscera. These tumors can start to grow in the retina, the brain, and in the spinal cord. Tumors also arise in the pancreas, epididymis, and the inner ear. If left untreated, this kind of disease can cause heart attacks, cardiovascular disease, or stroke. The age at which the tumors present ranges from childhood to the seventh decade of life.
08. Microcephaly. Microcephaly is a very rare neurological condition in which infant’s …show more content…
Paraneoplastic Pemphigus ( PNP ). The Pemphigus family is a rare blistering disease that affects the skin or the mucous membranes. This condition occurs in malignancies and is characterized by painful blisters and denuded areas of the mouth, lips, esophagus, and the skin. There are many forms of Pemphigus and Paraneoplastic Pemphigus is the least common and most serious. The disease is fatal as 90% of those who are diagnosed with the disease die because of sepsis, multi-organ failure, or cancer.
10.Morgellons. Morgellons disease is a mysterious, uncommon, and unexplained skin disease that is distinguished by crawling sensations, sores and fiber like filaments emerging from the sores. It is a relatively rare condition that frequently affects middle-aged women. Morgellons stands as a poorly understood disease and is believed to be a chronic infectious disease. There is no known cure or treatment for Morgellons.
Facts and statistics about genetic and rare …show more content…
Here are a few statistics and facts to illustrate the breadth of the 10 rare diseases problem worldwide.
1.There are estimated 7,000 types of rare disease and disorders
2.10% of the US population are living with a rare disease
3.Europe has approximately 30 million people living with a rare disease and 350 million people worldwide suffer from a rare disease.
4.If all the people with a rare disease will live in the same country, it will be the third world’s most populous country.
5.A condition is considered rare in the US if it affects fewer than 200,00 people combined.
6.80% of rare diseases are genetic and are present throughout the person’s life even if symptoms do not appear immediately.
7.50 % of the people affected by this disease are mostly children
8.30% of children with this disease will not be able to live to see their 5th birthday
9.35% of deaths in the first year of life are caused by this rare disease
10.The prevalence distribution of rare disease is skewed
11.95% of rare diseases have no FDA approved drug treatment
12.At the first 25 years of Orphan Drug Act, 326 new drugs were approved by the FDA for patients with a rare
08. Microcephaly. Microcephaly is a very rare neurological condition in which infant’s …show more content…
Paraneoplastic Pemphigus ( PNP ). The Pemphigus family is a rare blistering disease that affects the skin or the mucous membranes. This condition occurs in malignancies and is characterized by painful blisters and denuded areas of the mouth, lips, esophagus, and the skin. There are many forms of Pemphigus and Paraneoplastic Pemphigus is the least common and most serious. The disease is fatal as 90% of those who are diagnosed with the disease die because of sepsis, multi-organ failure, or cancer.
10.Morgellons. Morgellons disease is a mysterious, uncommon, and unexplained skin disease that is distinguished by crawling sensations, sores and fiber like filaments emerging from the sores. It is a relatively rare condition that frequently affects middle-aged women. Morgellons stands as a poorly understood disease and is believed to be a chronic infectious disease. There is no known cure or treatment for Morgellons.
Facts and statistics about genetic and rare …show more content…
Here are a few statistics and facts to illustrate the breadth of the 10 rare diseases problem worldwide.
1.There are estimated 7,000 types of rare disease and disorders
2.10% of the US population are living with a rare disease
3.Europe has approximately 30 million people living with a rare disease and 350 million people worldwide suffer from a rare disease.
4.If all the people with a rare disease will live in the same country, it will be the third world’s most populous country.
5.A condition is considered rare in the US if it affects fewer than 200,00 people combined.
6.80% of rare diseases are genetic and are present throughout the person’s life even if symptoms do not appear immediately.
7.50 % of the people affected by this disease are mostly children
8.30% of children with this disease will not be able to live to see their 5th birthday
9.35% of deaths in the first year of life are caused by this rare disease
10.The prevalence distribution of rare disease is skewed
11.95% of rare diseases have no FDA approved drug treatment
12.At the first 25 years of Orphan Drug Act, 326 new drugs were approved by the FDA for patients with a rare