Orphan Drugs: In Development

or infrequent ovulation (oligovulation).
in situ-Carcinoma in situ is tissue
cancerous only in its surface cells with
no sign of spread to deeper layers.
in vitro-Outside the body.
in vivo-Within the body.
IV-Intravenous.
Kaposi's sarcoma-Skin tumor that can
be accompanied by fever, enlarged lymph
nodes and gastrointestinal problems.
keratinization-Conversion of cells
into keratin, a tough fibrous protein that
is the main constituent of the outermost
layer of skin, nails and hair.
keratoconjunctivitis, vernal-Simultaneous inflammation of the cornea and
conjunctiva (transparent membrane
covering the white of the eye).
Keratoconjunctivitis sicca, or "dry
eye," is persistent corneal and conjunctival dryness caused by deficiency in tear
production. Can be caused by autoimmune
disorders, such as rheumatoid arthritis
and Sjogren's syndrome.
keratoplasty-The surgical transplantation of corneal tissue, or a "corneal graft."
lactic acidosis-Accumulation of excess
lactic acid in the blood, which can cause
hyperventilation and mental confusion
leading to stupor and coma.
Lambert-Eaton syndrome-Also called
myasthenic syndrome, in which a
defective release of acetylcholine (a neurotransmitter) in response to a nerve impulse
leads to muscle weakness. The syndrome
may be associated with small cell sarcoma
and some autoimmune diseases.
Lennox-Gastaut syndrome-Characterized by seizures and mental retardation
in infants and young children.
leprosy-Chronic infection by the bacterium Mycobacterium leprae (Hansen's
bacillus), which invades the nerves and
progresses to local invasion of other
tissue, such as skin, testis and lymph
nodes. Lepromatous leprosy is one of
two principal forms of the disease, characterized by diffuse skin lesions and, in
advanced cases, destructive lesions and
deformities. Treatment with the drug
dapsone sometimes causes erythema
nodosum leprosum, an eruption of
red-purple, extremely tender swellings
on the legs, usually accompanied by
fever and joint and muscle pains.
leukapheresis-The removal from a
blood donor of a quantity of white blood
cells, followed by return to the donor of
the remaining portions of the blood.
leukemia-A form of cancer involving
abnormally growing white blood cells,
which dominate the bone marrow and
prevent it from making enough normal
blood cells. This leaves the patient highly
susceptible to serious infections, anemia
and bleeding episodes. The cells increase
in the blood, interfering with the func
tion of other organs.
leukocyte-White blood cell.
levothyroxine-A synthetic version of
thyroxine, the most important of the

thyroid hormones.
lymphoma-Cancers in which the cells
of lymphoid tissue, found mainly in the
lymph nodes and spleen, multiply unchecked. Lymphomas fall into two categories: One is called Hodgkin's disease,
characterized by a particular kind of
abnormal cell. All others are called nonHodgkin's lymphomas, which vary in
their malignancy according to the nature
and activity of the abnormal cells. Band T-cell lymphomas are caused by
proliferation of the two principal types
of white blood cells, called B- and Tlymphocytes. Mycosis fungoides is a
type of lymphoma that primarily affects
the skin of the buttocks, back or shoulders
but can also occur in other sites. The
cause is unknown.
malaria-A serious parasitic disease, spread
by the bite of the Anopheles mosquito.
Malaria is characterized by severe fever
and chills, and complications affecting
the kidneys, liver, brain, and blood.
Plasmodium falciparum and plasmodium vivax are two of the four
species of the genus plasmodia (single- celled protozoa) responsible for the disease.
mastocytosis-Appearance of itchy,
irregular, yellow or orange-brown swellings on the skin, usually on the trunk.
melanoma-A cancer made up of
pigmented skin cells.
metastases-Secondary cancers that
have spread from the primary or original
cancer site.
methotrexate-An anticancer drug.
microinfusion-Infusion into a vein by
means of a fine, mechanically controlled
capillary tube.
multiple myeloma-A malignant condition characterized by the uncontrolled
proliferation and disordered function of
plasma cells (a type of white blood cell)
in the bone marrow. It occurs in middle
to old age and leaves patients vulnerable
to increased infections and anemia.
multiple sclerosis-Progressive disease
of central nervous system in which scattered patches of the covering of nerve
fibers (myelin) in the brain and spinal
cord are destroyed. Symptoms range
from numbness and tingling to paralysis
and incontinence.
muscular dystrophy-Inherited muscular disorder of unknown cause in
which muscle fibers slowly degenerate.
Duchenne MD is the most common type.
Mycobacterium avium disease-A
bacterial infection by the organism
Mycobacterium avium that can affect
most internal organs, resulting in widely
disseminated disease in AIDS patients. It
may be associated with symptoms such
as enlarged lymph nodes, weight loss
and diarrhea.
myelodysplastic syndromes-Blood

disorders that ultimately are fatal. Patients
usually succumb to infections or bleeding.
myoclonus-Rapid, uncontrollable
muscle jerks or spasms that occur at rest

or during movement. Can be caused by
nerve or muscle disease, epileptic
seizures or brain disorders.
myxedema coma-A rare comatose condition as a complication of severe hypothyroidism (underactivity of the thyroid
gland and underproduction of its hormones), most often seen in elderly women.
nephrolithiasis-The formation of, or
disease state characterized by, the presence
of kidney stones. (See calculi.)
neuropathy-Disease, inflammation, or
damage to the peripheral nerves, which
connect the central nervous system to
the sense organs, muscles, glands, and
internal organs.
neutropenia-Abnormally low count of
certain white blood cells, leaving a patient
vulnerable to bacterial and fungal infections.
osteomyelitis-Infection of bone and
bone marrow, usually by bacteria.
Paget's disease-Also called osteitis
deformans, a bone disorder of unknown
cause in which the normal process of
bone formation is disrupted, causing
bones to weaken, thicken and become
deformed. It commonly affects the pelvis,
skull, spine and long bones of the leg
palliative treatment-A treatment that
relieves symptoms but does not cure.
papillomavirus/papillomatosis-The
papillomavirus is the viral agent of warts,
believed to be contagious and mostly
harmless, affecting only the skin's topmost layer. Laryngeal papillomatosis
is characterized by lesions of the larynx
thought to be caused by the papillomavirus. Patients experience hoarseness that
progresses to a noisy, high-pitched
breathing sound.
parenteral-The administration of drugs
or other substances by any route other
than via the gastrointestinal tract, e.g., by
injection into a blood vessel or a muscle.
Parkinson's disease-Chronic neurologic
disease of unknown cause, characterized
by tremors, rigidity and an abnormal
gait. The most common variety is idiopathic Parkinson's disease, or paralysis
agitans. Post-encephalitic parkinsonism is the disease-like state occurring
after cases of encephalitis, an inflammation of the brain.
penicillamine-An anti-rheumatic drug
also used to treat copper, mercury, lead
or arsenic poisoning.
Pneumocystis carinii pneumonia
(PCP)-Severe lung infection caused by
the parasite Pneumocystis carinii. PCP is
found in nearly 80% of all AIDS patients
at some time during the course of the
disease and is a major cause of death in
these patients.
polycystic ovarian disease-A condition characterized by scanty, or absence
of, menstruation, infertility, excessive
facial hair and obesity. The ovaries con
tain multiple cysts. One theory postulates
that the disease is due to an imbalance in
the pituitary in the ratio of gonadotropic
hormones-the LH/FSH ratio.

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