Rare diseases are any diseases that affects a small percentage of the population.
They are characterized by a wide diversity of symptoms and signs that vary not only from disease to disease but also from patient to patient suffering from the same disease.
Most rare diseases are genetic and are present throughout a person’s entire life, even if the symptoms do not appear at birth.
Examples of some rare diseases
1. Huntington disease
Huntington disease is a progressive and inherited fatal brain disorder inherited that gradually kills off healthy nerve cells in the brain.
It leads to loss of language, thinking and reasoning abilities, memory, coordination and movement.
It has no cure.
2. Amyotrophic lateral sclerosis (ALS)
Amyotrophic lateral sclerosis also known as Lou Gehrig’s disease) is a group of rare neurological diseases that mainly involve the nerve cells (neurons) responsible for controlling voluntary muscle movement.
Voluntary muscles produce movements like chewing, walking, and talking. The disease is progressive, meaning the symptoms get worse over time.
Currently, there is no cure for ALS and no effective treatment to halt, or reverse, the progression of the disease.
3. Muscular dystrophy
Muscular dystrophy is a group of diseases that cause progressive weakness and loss of muscle mass.
In muscular dystrophy, abnormal genes (mutations) interfere with the production of proteins needed to form healthy muscle. It has no cure.
There are nine major forms of muscular dystrophy
4 .Acquired aplastic anemia
This is a serious blood disorder, due to failure of the bone marrow to produce blood cells.
Bone marrow is the spongy substance found in the center of the bones of the body, in adults mainly the spine, pelvis, and large bones of the legs.
Red and white blood cells and platelets are formed in the bone marrow. The cells are released into the bloodstream to travel throughout the body performing their specific functions.
Red blood cells deliver oxygen to the body’s organs, white blood cells help in fighting infections, and platelets form clots to stop bleeding.
5. Guillain-Barré syndrome
This is a serious autoimmune disorder in which the immune system attacks healthy nerve cells in your peripheral nervous system.
This leads to weakness, numbness, and tingling. It can eventually cause paralysis.
Guillain-Barré syndrome may be triggered by an acute bacterial or viral infection.
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