Recommended Drug Therapy

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Autoimmune Disorders

The immune system is a complicated network of cells and cell components (called molecules) that normally work to defend the body and eliminate infections caused by bacteria, viruses, and other invading microbes. If a person has an autoimmune disease, the immune system mistakenly attacks itself, targeting the cells, tissues, and organs of a person's own body. A collection of immune system cells and molecules at a target site is broadly referred to as inflammation.

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Gaucher Disease

Gaucher Disease is the most common lipid-storage disorder, and is the most common genetic disease affecting Jewish people of Eastern European ancestry. The disease results from a specific enzyme deficiency in the body, caused by a genetic mutation received from both parents. Its course is quite variable, ranging from no outward symptoms to severe disability and death.

Lysosomal Storage Diseases

Lysosomal storage diseases are caused by a lack of enzymes that normally eliminate unwanted substances in the cells of the body. The enzymes are found in sac-like structures in cells called lysosomes. Lysosomes act as the "recycling center" of each cell, breaking down unwanted material into simple products for the cell to use to build new material. The lack of certain enzymes causes a buildup of the substance that the enzyme would normally eliminate, and deposits accumulate in many cells of the body. Abnormal storage causes inefficient functioning and damage of the body's cells, which can lead to serious health problems.

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Multiple Sclerosis (MS)

MS is thought to be an autoimmune disease that affects the central nervous system (CNS). The CNS consists of the brain, spinal cord, and the optic nerves. Surrounding and protecting the nerve fibers of the CNS is a fatty tissue called myelin, which helps nerve fibers conduct electrical impulses.
 
In MS, myelin is lost in multiple areas, leaving scar tissue called sclerosis. These damaged areas are also known as plaques or lesions. Sometimes the nerve fiber itself is damaged or broken. Myelin not only protects nerve fibers, but makes their job possible. When myelin or the nerve fiber is destroyed or damaged, the ability of the nerves to conduct electrical impulses to and from the brain is disrupted, and this produces the various symptoms of MS.

Parkinson's Disease

Parkinson's disease occurs when a group of cells, in an area of the brain called the substantia nigra, that produce a chemical called dopamine begin to malfunction and eventually die. Dopamine is a neurotransmitter, or chemical messenger, that transports signals to the parts of the brain that control movement initiation and coordination. When Parkinson's disease occurs, for unexplained reasons, these cells begin to die at a faster rate and the amount of dopamine produced in the brain decreases.

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Plaque Psoriasis

Plaque psoriasis is the most typical form of the disease—four out of five people with psoriasis have this type. The technical name for plaque psoriasis is "psoriasis vulgaris" (vulgaris means common). Typically, it is characterized by patches on the elbows, knees, scalp and lower back, but it can be found on any area of the skin.

Respiratory Syncytial Virus (RSV)

Respiratory Syncytial Virus (RSV) is the leading cause of lower respiratory tract infections in infants and young children. Approximately two-thirds of infants are infected with RSV during the first year of life and almost 100% have been infected by age two.  Most of these RSV infections cause minor upper respiratory illness.  However, in certain high-risk pediatric patients, RSV infection may cause serious lower respiratory tract disease.

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