Rare Diseases

Metabolic Disorders
The foods we eat every day are filled with the building blocks of life: carbohydrates, amino acids, and fats. But to use these materials, the body must break down complex foods...
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Rare Blood Pressure Disorders
A person's blood pressure is continually changing depending on activity, temperature, diet, emotional state, posture, physical state, and medication use... Read more>

Crohn's Disease
For patients with Crohn's disease, attacks can bring everyday activities to a screeching halt as the incurable, inflammatory bowel disease ravages their gastrointestinal tract... Read more>

Interstitial Cystitis
Imagine needing to run to the bathroom up to 60 times a day. You might have to get up five times in the middle of dinner at a restaurant or wake up every 25 minutes...
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Hyperparathyroidism
Cinacalcet is the first in a new class of drugs known as calcimimetics, now available to patients whose parathyroid glands are producing too much parathyroid hormone...
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Acromegaly
When a manufactured protein became available in 2003 for people with acromegaly, it marked the first in a new class of drugs that block the effect of the growth hormone... Read more>

Diseases of the Developing World
Some diseases considered rare in the United States are actually very common around the world. For these diseases, new treatments being developed in the United States...
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Blood Disorders
Our bodies depend on small, specialized cells that make up blood to transport nutrients, gases, and waste products throughout our bodies. An imbalance of too many or too few... Read more>

Leprosy
Leprosy is an infectious disease caused by the bacteria Mycobacterium leprae with symptoms of disfiguring skin lesions and sensory loss in the skin... Read more>

Childhood Diseases
Premature babies face an uphill battle as their fragile, still-developing systems fight to overcome many issues... Read more>

Narcolepsy
In people with narcolepsy the brain does not properly regulate sleep and wake cycles, so they experience episodes of frequent, uncontrollable daytime sleeping... Read more>

ALS/Lou Gehrig's Disease
Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's Disease, is a progressive disease that affects motor nerve cells in the brain and spinal cord... Read more>

Cancer
Cancer is a group of diseases in which abnormal cells develop and spread through parts of the body. Anyone can develop cancer... Read more>

Leukemia
In leukemia, the bone marrow produces abnormal white blood cells—the leukemia cells, which in time may crowd out normal white blood cells, red blood cells, and platelets... Read more>