Rare diseases are also called “orphan” diseases. This means that so few people get them that there’s very little research on how to cure them.
Multiple Sclerosis
About 90 out of 100,000 people will get Multiple Sclerosis or MS. This autoimmune disease happens when your body starts to attack the insulation around nerve cells. Like the plastic that is around electrical wires, losing this outer layer means neurological issues and eventually, death. There are treatments out there to slow down its progression, but no cure.
Narcolepsy
Narcolepsy is a sleep disorder where people have trouble staying awake. Only about 50 people in 100,000 are diagnosed with narcolepsy each year. There are certain drugs that target the sleep centers of the brain. But currently, there is no cure.
Primary biliary cholangitis
Primary biliary cholangitis damages the bile ducts. These are the tubes that carry bile from the liver to the gallbladder. Eventually, the bile ducts are destroyed, which leads to liver scarring, digestive issues, and cirrhosis. There is no cure and in severe cases, patients require a liver transplant.
Fabry disease
Fabry disease is a genetic disorder that affects 30 out of 100,000 people in the United States. Those who have it lack an enzyme that breaks down certain fats. Over time, this fat accumulates in cells, damaging them. Other affects are heart attack, stroke, and kidney disease.
Cystic fibrosis
Twenty-five out of 100,000 children are diagnosed with cystic fibrosis. This is another genetic disorder and it causes thick mucus to build-up in the lungs and the digestive tract. There is no cure, but advances in gene therapy and treatment of antibiotic-resistant bacterial infections have prolonged the lives of cystic fibrosis sufferers.
