Cystic syndromal

PA S1 S01.131 Neutrophil elastase Drug target for emphysema, cystic fibrosis, adult respiratory distress syndrome, rheumatoid arthritis and other diseases... 

Andreoli TE Ion transport disorders introductory comments. Am J Med 1998 104 85. (First of a series of articles on ion transport disorders published between January and August, 1998. Topics covered were structure and function of ion channels, arrhythmias and antiarrhythmic drugs, Liddle syndrome, cholera, malignant hyperthermia, cystic fibrosis, the periodic paralyses and Bartter syndrome, and Gittelman syndrome.)... 

Sepsis syndrome Anorexia nervosa Complications during pregnancy Geriatric patients with multiple chronic disease Organ transplantation Inborn errors of metabolism Cystic fibrosis Extreme prematurity... 

Damage to connective caused by leakage of elastases leads to damage associated with inflammatory diseases, such as pulmonary emphysema, adult respiratory distress syndrome, septic shock, cystic fibrosis, carcinogenesis, chronic bronchitis, and rheumatoid arthritis. Compounds that directly inhibit elastase or its release from human neutrophils are of enormous pharmaceutical and cosmetological interest in the development of new anti-inflammatory drugs. A possible source for elastase inhibitors are the medicinal Asteraceae and Droseraceae, particularly those used as traditional medicine in Asia. 

Androgen AR Human cell line Cancer, andropause, menopause, Sjogren s syndrome, idiopathic hirsutism, infertility, hypertension, mood disorders, diabetes, cystic fibrosis Immune modulation, vascular hemodynamics, neuroprotection, skeletal development, reproduction, maintenance of neuronal morphology, regulation of vasopressin Vla expression, prostatic blood flow and LH production... 

Currently, there is stUl a gap for the potential of gene therapy to be fulfilled. Gene therapy clinical trials have been conducted for diseases such as severe combined immunodeficiency disease (SCID, bubble baby syndrome), sickle cell anemia, cystic fibrosis, familial hypercholesterolemia, and Gaucher disease. 

While certain behavioral and nonbehavioral diseases are believed to be monogenic, diseases such as Huntington s, cystic fibrosis, Marfan, and Hirschsprung result in the specified disease, and the outward appearance or result (phenotype) of the disease varies between individuals. For instance, for Marfan syndrome, there is a level below which the mutant protein does not exhibit itself in an outward manner. Most of these diseases have modifier genes that cause modifications in the outward demonstration of the disease and play a key role in the clinical symptoms. Further, the particular metabolic pathways are often varied, with several of the steps being important and the importance of each mechanistic pathway may differ with every individual. 

Gl obstruction (Concerts only) Because the Concerts tablet is nondeformable and does not appreciably change shape in the Gl tract, do not administer to patients with pre-existing severe Gl narrowing (eg, small bowel inflammatory disease, short gut syndrome because of adhesions or decreased transit time, history of peritonitis, cystic fibrosis, chronic intestinal pseudo-obstruction, Meckel s diverticulum). 

The regular digestion of dietary fats and fat-soluble substances and/or their absorption via the lipid route is compromised in the frame of various diseases such as cystic fibrosis, short bowel syndrome, cholestasis, and chronic inflammatory bowel diseases. 

As discussed above, in the case of phenylketonuria, early intervention can make the difference between mental retardation and a near normal life course for a newborn. Congenital adrenal hyperplasia and maple syrup urine disease are two examples of neonatal hereditary disorders where early diagnosis and medical intervention can make the difference between life and death for the newborn. In addition, in a number of genetic diseases, early diagnosis and treatment can help ameliorate symptoms these include fragile X syndrome, homocystinuria, sickle cell anemia, cystic fibrosis, and many /1-thalassemias. 

Extreme clinical examples of androgen excess include central precocious puberty, the adrenogenital syndromes, and androgen-secreting adrenal, ovarian, or testicular tumors. Less severe problems include idiopathic hirsutism, premenstrual syndrome, and severe cystic acne. 

Unlabeled Uses Treatment of edema associated with CHF, liver cirrhosis, and nephrotic syndrome treatment of hypertension reduces lithium-induced polyuria, slows pulmonary function reduction in cystic fibrosis... 

Pancreatic enzyme replacement or supplement when enzymes are absent or deficient, such as with chronic pancreatitis, cystic fibrosis, or ductal obstruction from cancer of the pancreas or common bile duct to reduce malabsorption treatment of steatorrhea associated with bowel resection or postgastrectomy syndrome PO 1-3 capsules ortablets before or with meals or snacks. May increase to 8 tablets/dose. 

Pediatric patients who develop psychiatric syndromes in association with chronic illness (e.g., major depression in a patient with cystic fibrosis)... 

The causes of human copper deficiency include (1) low intake - malnutrition, total parenteral nutrition (TPN) (2) high loss - cystic fibrosis, nephrotic syndromes and (3) genetic factors — Menkes disease. Copper deficiency may also be associated with chronic malabsorption, a situation which is made much worse in cases of gastric and bowel resection. Several special diets, including powdered milk, liquid protein and standard hospital diets are a means of inducing copper deficiency. The amount of copper in US food has decreased steadily since 1942, and may be related to the rising incidence of coronary artery disease. A copper deficiency may also occur as the result of the use of chelators for other purposes for example, diethyl dithiocarbamate is an in vivo metabolite of ANTABUSE (disulfiram). 

OHSS is characterized by cystic ovarian enlargement, increased capillary permeability, and third space fluid accumulation (that is in an extracellular compartment that is not in equilibrium with either the extracellular or intracellular fluid, for example the bowel lumen, subcutaneous tissues, retroperitoneal space, or peritoneal cavity). Risk factors include a previous history of OHSS, age under 30 years (probably because more follicles are available), and polycystic ovary syndrome. Non-pregnant patients usually recover within 14 days with supportive treatment. The severe form (with ascites or pleural effusion and hemoconcentration) occurs in 1-10% of patients (64,65). In critical cases, hypoxemia, renal insufficiency, thromboembolism, and rarely death can occur (66). 

F. W., McGhee, J. R., Kelly, D., Lyrene, R. K. and Sorscher, E. J. (2001). A clinical inflammatory syndrome attributable to aerosolized lipid-DNA administration in cystic fibrosis. Hum. Gene Ther. 12, 751-761. 

Some congenital diseases such as Marfan syndrome or Ehlers Danlos syndrome (type 4) can cause arteriopathies of cranial cervical vessels. In Ehlers Danlos syndrome, elongations, dissections, dilatation and aneurysms, as well as fistula in large and mid-size arteries, are found. Cystic medial necrosis (Ueda et al. 1999) leads to aortic dissections which can include supraaortic vessels (Fig. 5.27)... 

Single-gene (also called Mendefian) disorders number more than 6000, with an incidence of about 1 in 200 births. Some examples include cystic fibrosis, sickle ceU anaemia, Marfan syndrome, Huntington s disease and hereditary haemochromatosis. Single-gene disorders are inherited in recognisable patterns autosomal dominant, autosomal recessive and X-hnked. 

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