Pegademase
Article Abstract:
Pegademase is a drug made by conjugating the bovine enzyme adenosine deaminase (ADA) with polyethylene glycol (PEG). This drug has recently been approved by the US Food and Drug Administration as an 'orphan drug,' a therapeutic drug that is not profitable for manufacturers to produce. This drug was approved for the treatment of severe combined immunodeficiency disease (SCID), which is very rare (less than one per million births) and is characterized by an inherited deficiency of ADA. Without this enzyme, toxic substances build up in lymphocytes (white blood cells) and kill them, leading to a lymphocyte deficiency and an increased susceptibility to infection. Before pegademase became available, the treatment of choice for these children was a bone marrow transplant. The patients have had to be severely isolated, and until recently, often died before the age of two. Pegademase is given by injection into muscle tissue once or twice each week. Fourteen children were treated in clinical trials of the drug and T-lymphocyte counts increased in all patients within a few weeks to six months from initiating treatment. Most of these children have recovered specific immune responses; a few have not responded as quickly or completely. All of the children showed improvements in their ability to combat infections, and also had fewer infections. Pegademase must be given indefinitely, as it does not cure SCID. The cost for one year's dosage for one child is currently about $60,000. The long-term effectiveness of the drug is not known at this time, and no adverse effects have been reported. (Consumer Summary produced by Reliance Medical Information, Inc.)
Publication Name: Medical Letter on Drugs and Therapeutics
Subject: Health
ISSN: 0025-732X
Year: 1990
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Betaine for homocystinuria
Article Abstract:
The orphan drug betaine (Cystadane) may be effective in reducing plasma homocystine concentration in homocystinuria, a genetic disease characterized by metabolic abnormalities. Minimal side effects of the drug are restricted to nausea and diarrhea. Long-term therapy does not seem to be associated with a risk of thromboembolism, a common aspect in homocystinuria patients. Decreasing the level of homocystine in blood may reduce cardiovascular risk in men.
Publication Name: Medical Letter on Drugs and Therapeutics
Subject: Health
ISSN: 0025-732X
Year: 1997
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Sodium phenylbutyrate for urea cycle enzyme deficiencies
Article Abstract:
Sodium phenylbutyrate (Buphenyl) therapy in combination with restricted protein intake and amino acid supplementation may prolong life in patients with urea cycle disorders, a rare liver enzyme deficiency. The orphan drug phenylbutyrate can also preserve the cognitive functions in late-onset disease. Menstrual abnormalities are a frequent side effect, while loss of appetite, body odor, or changes in taste are quite rare.
Publication Name: Medical Letter on Drugs and Therapeutics
Subject: Health
ISSN: 0025-732X
Year: 1996
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