Cerebral Palsy

Cerebral palsy (CP) is an umbrella-like term used to describe a group of chronic disorders that impair control of movement. These disorders appear in the first few years of life and generally do not worsen over time. The term cerebral refers to the brain’s two halves, or hemispheres, and palsy describes any disorder that impairs control of body movement. Faulty development or damage to motor areas in the brain disrupts the brain’s ability to adequately control movement and posture.

Commonly Prescribed (On-Label) Drugs: None

Off-Label Prescription Drugs Breakthrough Options: Botulinum Toxin Type A, Glycopyrrolate

Cystic Fibrosis

Cystic fibrosis (CF) is an inherited condition affecting the cells that produce mucus, sweat, saliva, and digestive juices. Normally, these secretions are thin and slippery, but in CF, a defective gene causes the secretions to become thick and sticky. Respiratory failure is the most dangerous consequence of CF. Each year approximately 3,200 Caucasian babies are born in the United States with CF. The disease is much less common among African-American and Asian-American children. Most babies born with CF are diagnosed by age three, although mild forms of the disease may not be detected until the third, fourth or fifth decade of life.

Commonly Prescribed (On-Label) Drugs: Acetylcysteine, Aztreonam, Ceftazidime, Chloramphenicol, Dornase Alfa, Fusidic Acid, Medium Chain Triglycerides, Pancrelipase, Ticarcillin/Clavulanic Acid, Tobramycin, Ursodiol

Off-Label Prescription Drugs Breakthrough Options: Dexamethasone, Fluticasone, Prednisolone, Prednisone, Theophylline

Juvenile Rheumatoid Arthritis

Juvenile rheumatoid arthritis (JRA) is arthritis that causes joint inflammation and stiffness for more than six weeks in a child of 16 years of age or less. Inflammation causes redness, swelling, warmth, and soreness in the joints, although many children with JRA do not complain of joint pain. Any joint can be affected and inflammation may limit the mobility of affected joints. One type of JRA can also affect the internal organs. Doctors classify JRA into three types by the number of joints involved, the symptoms, and the presence or absence of certain antibodies found by a blood test.

Commonly Prescribed (On-Label) Drugs: Aurothioglucose, Choline Salicylate

Off-Label Prescription Drugs Breakthrough Options: Anakinra, Auranofin, Diclofenac, Infliximab, Methylprednisolone, Nabumetone

Kawasaki Syndrome

Kawasaki disease is a rare condition in children (usually between the ages of 2 and 5) that involves inflammation of the blood vessels. The condition is usually accompanied by a fever that lasts at least five days. Other classic symptoms may include: red eyes, lips and mouth; a rash; swollen red hands and feet; and swollen lymph nodes.

Commonly Prescribed (On-Label) Drugs: Immune Globulin IV

Off-Label Prescription Drug Breakthrough Option: Infliximab

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