Orphan Drugs: In Development

Glossary acne rosacea-A chronic face disorder characterized by flushing, burning and stinging, and inflammatory papules or pustules. It affects about 1 in 500 people, most commonly middle-aged women. acquired immune deficiency syndrome (AIDS)-A manifestation of infection with the human immunodeficiency virus (HIV) characterized by the presence of one or more opportunistic diseases which occur primarily, or in some cases only, when an individual's immune system function is defective. adenocarcinoma-Cancer of glandular tissue, or tumor of which gland-derived cells form glandlike structures. adenosine deaminase deficient (ADA deficiency)-Inherited enzyme deficiency disease in newborns that results in a partial or total dysfunction of the immune system, leaving newborns unable to resist common infections. adjunct-An auxiliary treatment that is secondary to the main treatment. adjuvant-A substance or drug that aids another substance in its action. agonist-A drug that competes with a nerve receptor producing a typical response. alpha 1-proteinase inhibitor deficiency-Although it is a rare condition, some people are congenitally deficient in alpha 1-proteinase inhibitor (or alpha 1-trypsin, a glycoprotein), which predisposes them to pulmonary emphysema early in life, even in the absence of exposure to substances (like cigarette smoke) that interfere with lung-defense mechanisms. amenorrhea-Absence of cyclic menstrual periods, often due to failure to ovulate. Hypothalamic amenorrhea is caused by hypogonadotropic hypogonadism-deficiency or absence of pulsatile gonadotropin-releasing hormone (GnRH), which stimulates cell activity in the ovaries. Gonadotropin therapy refers to treatment with GnRH to induce ovulation and aid fertility. (Blockage of Fallopian tubes-bilateral tubal occlusion-also causes infertility.) amyotrophic lateral sclerosis (ALS)Also known as Lou Gehrig's disease. It is the most common of the motor neuron diseases, a group of rare disorders in which the nerves that control muscular activity degenerate within the brain and spinal cord causing weakness and wasting of the muscles. anorexia-Prolonged loss of appetite that leads to significant weight loss. antithrombin III (AT-III) deficiencyToo little AT-III enzyme, which neutralizes factors that coagulate blood, can cause thrombosis (blood clot within an intact blood vessel), pulmonary embolism (blood clot obstructing pulmonary artery) and thromboembolism (fragment of thrombosis that has traveled to another place in the body). antithrombotic-A drug that breaks down blood clots that have formed in the body. aplastic anemia-Caused by failure of the bone marrow to produce red blood cells. Condition can be fatal if left untreated. application submitted-An application for marketing has been submitted by the sponsor to the Food and Drug Administration. attenuation-A reduction in the severity or virulence of a disease or organism. bacteremia-Presence of bacteria in the blood stream. Bacteria (anaerobic) causing gram-negative bacteremia release poison after they die, produce fever, and cause fluid to leak from more permeable walls of blood capillaries into surrounding tissues. Endotoxic shock, a serious drop in blood pressure, can result. B-cell-A class of white blood cells important to the body's immune system. BCNU-A generic anticancer drug, also known as carmustine. blepharospasm-Involuntary, prolonged contraction of an eyelid muscle that causes almost complete eye closure. buccal cavity-The cavity formed by the cheeks and mouth. cachexia-Profound state of general ill health characterized by malnutrition, weakness and emaciation. calculi, renal and bladder-Stones formed or present in the kidneys or bladder caused by dissolved substances in the urine. Apatite, struvite, and uric acid varieties refer to specific composition of the calculi. cancer, germ cell-Localized proliferation of cells that are immature forms of either sperm or ova, which can produce abnormal levels of alpha-fetoprotein (AFP) (normally produced in the fetus). Trophoblastic cell tumors arise from tissues that form the placenta and can produce human chorionic gonadotropin (HCG) (a hormone normally produced by the placenta in early pregnancy and essential to a healthy pregnancy). Both AFP and HCG can be monitored to assess treatment or recurrence of tumors. carcinoma-Cancer. Invasive carcinoma has spread, or has the tendency to spread, throughout body tissues. Squamous cell carcinoma is one of the three most common types of skin cancer, arising from the flattened, scalelike cells in the skin and resulting primarily from long-term exposure to the sun. carnitine deficiency-Lack of a particular constituent in muscle and liver tissue, causing tissue to use oxygen inefficiently. catabolism-Metabolism involving the release of energy, resulting from the breakdown of complex materials. causalgia-Persistent burning pain, usually in a limb, resulting from a penetrating nerve injury. The sympathetic nervous system controls many involuntary activities of the body, including caliber of blood vessels and actions of nerve cells in response to stimuli (reflexes). Restriction of nerve cell activity or blood vessels causes inadequate nutrition of tissues, resulting in reflex sympathetic dystrophy. cerebral palsy-A general term for disorders of movement and posture resulting from damage to the brain in pregnancy, during birth, in the newborn period, or in early childhood. cervical dystonia-Disorder or lack of muscle tone in the muscles of the neck. Charcot-Marie-Tooth disease-An inherited muscle-wasting disease that mainly affects the legs. It is more common in boys and usually appears in late childhood or adolescence. colitis-Inflammation of the colon (large intestine) causing diarrhea, usually vwith blood and mucous, and sometimes with abdominal pain and fever. Colitis may be due to a variety of causes, including infection by a virus, amoeba, or a bacterium that produces toxins that irritate the intestinal lining. Antibiotic therapy for more than two weeks may result in a form of colitis caused by the bacterium Clostridium difficile, which produces an irritating toxin. This toxin causes the lesion of pseudomembranous enterocolitis, which is characterized by profuse watery diarrhea with cramps and low-grade fever. Ulcerative colitis is chronic inflammation and ulceration of the lining of the colon and rectum. It causes bloody diarrhea and mainly involves the left colon. corneal disease-Disorders affecting the cornea, a transparent thin-walled dome forming the front of the eyeball. A corneal ulcer is a break, erosion or open sore in the outer layer (epithelium), which sometimes extends into the middle layer. Squamous metaplasia refers to transformation of the flat cells of the ocular surface epithelia, which consists of the cornea and the conjunctivathe mucous membrane covering the white of the eye. cryptococcal meningitis-A fungal infection that affects the three membranes (meninges) surrounding the brain and spinal cord. Symptoms include severe headache, vertigo, nausea, anorexia, sight disorders and mental deterioration. Cryptosporidium parvum-The protozoan parasite that causes cryptosporidial diarrhea, characterized by chronic, profuse, watery diarrhea accompanied by fever, marked weight loss and enlarged lymph nodes. 19

/ 24

Actions

file_download Download Options Download this page PDF - Pages #1-24 Image - Page 19 Plain Text - Page 19

About this Item

Title
Orphan Drugs: In Development
Author
Pharmaceutical Research and Manufacturers of America
Canvas
Page 19
Publication
Pharmaceutical Manufacturers Association.
1992
Subject terms
reports
Item type:
reports

Technical Details

Link to this Item
https://name.umdl.umich.edu/5571095.0504.010
Link to this scan
https://quod.lib.umich.edu/c/cohenaids/5571095.0504.010/19

Rights and Permissions

The University of Michigan Library provides access to these materials for educational and research purposes, with permission from their copyright holder(s). If you decide to use any of these materials, you are responsible for making your own legal assessment and securing any necessary permission.

Manifest
https://quod.lib.umich.edu/cgi/t/text/api/manifest/cohenaids:5571095.0504.010

Cite this Item

Full citation
"Orphan Drugs: In Development." In the digital collection Jon Cohen AIDS Research Collection. https://name.umdl.umich.edu/5571095.0504.010. University of Michigan Library Digital Collections. Accessed June 10, 2025.
Do you have questions about this content? Need to report a problem? Please contact us.