Glossary of Terms - Arthritis

application submitted— An application for marketing has been submitted by the company to the Food and Drug Administration (FDA).

 

Duchenne muscular dystrophy— An inherited disorder that involves rapidly worsening muscle weakness. Other muscular dystrophies get worse much more slowly. Duchenne muscular dystrophy is caused by a defective gene. Because of the way the disease is inherited, males are more likely to develop symptoms than are women.

 

fibromyalgia— Fibromyalgia syndromes are a group of disorders of unknown cause characterized by achy pain and stiffness in soft tissues, including muscles, tendons and ligaments. Fibromyalgia (pain and stiffness) may occur throughout the body or may be restricted to certain locations. A common variation, the primary fibromyalgia syndrome, usually occurs in pre- viously healthy young women who may be depressed, anxious, or stressed, often with interrupted and nonrestorative sleep.

 

Friedreich’s ataxia— An inherited disease that causes progressive damage to the nervous system resulting in symptoms ranging from gait disturbance and speech problems to heart disease. “Ataxia,” which refers to coordination problems such as clumsy or awkward movements and unsteadiness, occurs in many different diseases and conditions.

 

gout— The pain of gout (called attacks or flares) is caused by inflammation when needle-like crystals are deposited in connective tissue and/or in the fluid that cushions a joint (the synovial fluid). These crystals are made up of uric acid, a substance produced when the body breaks down purines found in human tissue and many foods we eat. Most uric acid is carried through the bloodstream to the kidneys, which eliminate it from the body in the urine. However, if the body produces too much uric acid or if the kidneys don’t eliminate enough of it, uric acid can build up in the blood. An attack usually starts with sudden, severe pain, tenderness, redness, warmth, and swelling in the large joint of the big toe. After about 3-10 days, the attack usually subsides, and the next one may not happen for months or even years. But over time, the gout attacks can become more severe, last longer, affect more than one joint, and occur more often.

 

muscular atrophy— The wasting or loss of muscle tissue.

 

muscular spasticity— An abnormal increase in muscle tone. It may be associated with involuntary muscle spasms, sustained muscle contractions, and exaggerated deep tendon reflexes that make movement difficult or uncontrollable. Although it most commonly affects the legs and arms, spasticity can affect any part of the body including the trunk, neck, eyelids, face, or vocal cords.

 

osteoarthritis— The most common form of joint disease, characterized by degeneration of the cartilage that lines joints and by the formation of reactive bony outgrowths at the boundary of a joint.

 

osteonecrosis— Bone death caused by poor blood supply to the area, most commonly in the hip and shoulder. After a while, the bone can break off. If this condition is not treated, bone damage gets worse, and eventually, the affected part of the bone may collapse. Osteonecrosis can be caused by disease, or a severe trauma, such as a break or dislocation, that affects the blood supply to the bone. Many times, no trauma or disease is present. That is called “idiopathic osteonecrosis,” which means it occurs without any known cause.

 

osteopenia— Refers to bone mineral density (BMD) that is lower than normal peak BMD but not low enough to be classified as osteoporosis. Bone mineral density is a measurement of the level of minerals in the bones, which shows how dense and strong they are. Having osteopenia means there is a greater risk that, as time passes, you may develop osteoporosis.

 

osteoporosis—The most common metabolic bone disease in older people in which a reduction in bone mass leads to fractures, especially of the vertebrae, hips and wrists.

 

Phase 0— First-in-human trials conducted in accordance with FDA’s 2006 guidance on exploratory Investigational New Drug (IND) studies designed to speed up development of promising drugs by establishing very early on whether the agent behaves in human subjects as was anticipated from preclinical studies.

 

Phase I— Safety testing and pharmacological profiling in humans.

 

Phase II— Effectiveness and safety testing in humans.

 

Phase III— Extensive clinical trials to demonstrate safety and efficacy in humans.

 

psoriatic arthritis— A type of chronic inflammatory arthritis associated with the chronic skin condition psoriasis, which is believed to be an inherited condition. Psoriatic arthritis typically appears about 10 years after the onset of psoriasis, which can manifest at any age, though most people are diagnosed between the ages of 15 and 35.

 

Raynaud’s disease— A condition that causes some areas of the body— fingers, toes, the tip of the nose, and ears—to feel numb and cool in response to cold temperatures or stress. In Raynaud’s disease, the smaller arteries that supply blood to the skin narrow, thus limiting blood circulation to affected areas.

 

rheumatoid arthritis— A type of arthritis that particularly attacks the small joints of the hands, wrists and feet. The joints become painful, swollen, stiff and, in severe cases, deformed.

 

scleroderma— A group of rare, progressive diseases that involve the hardening and tightening of the skin and connective tissues—the fibers that provide the framework and support for the body. Localized scleroderma affects only the skin. Systemic scleroderma also harms internal organs, such as the heart, lungs, kidneys, and digestive tract.

 

Sjögren’s syndrome— A chronic autoimmune disease in which a person’s white blood cells attack their moisture-producing glands. Although the hallmark symptoms are dry eyes and dry mouth, Sjögren’s may also cause dysfunction of other organs such as the kidneys, gastrointestinal system, blood vessels, lungs, liver, pancreas, and the central nervous system. Patients may also experience extreme fatigue and joint pain and have a higher risk of developing lymphoma.

 

systemic lupus erythematosus— A chronic autoimmune disorder, affecting many systems of the body, including the kidneys and brain.