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ERT was not used in clinical practice until 1991, after the FDA gave orphan drug approval for the treatment of Gaucher disease with Alglucerase. [1] ERTs were initially manufactured by isolating the therapeutic enzyme from human placenta. [1]
Many other types of drug treatments may require a titration or stepping up phase. This strategy is used to build tolerance to adverse events or to reach a desired clinical effect. [ 21 ] This also prevents accidental overdose and is therefore recommended when initiating treatment with drugs that are extremely potent and/or toxic (drugs with a ...
[4] [5] Side effects when used as drugs may include loss of appetite, nausea, vomiting, loose stools, vivid dreams at night, dehydration, rash, bradycardia, peptic ulcer disease, seizures, weight loss, rhinorrhea, salivation, muscle cramps, and fasciculations. [6] [7] ChEIs are indirect-acting parasympathomimetic drugs. [8]
It is an antitumor and antiangiogenic agent with oral bioavailability. Inhibition of TACE and MMP-1 are linked to the musculoskeletal side effects seen in hydroxamate metalloproteinase inhibitors, but this compound spares the enzymes. [12] It has been shown to diminish tumor growth in nasal cancer in rats as well as prostate cancer cell cultures.
Common side effects include vomiting, abdominal pain, constipation, and diarrhea. [3] Other side effects include perianal irritation and high blood uric acid. [5] The enzymes are from pigs. [5] Use is believed to be safe during pregnancy. [5] The components are digestive enzymes similar to those normally produced by the human pancreas. [6]
In the past twenty years, new medications, enzyme replacement, gene therapy, and organ transplantation have become available and beneficial for many previously untreatable disorders. Some of the more common or promising therapies are listed: [citation needed]
Methylene blue (methylthioninium chloride), the antidote indicated for drug-induced methemoglobinemia on the World Health Organization's List of Essential Medicines, among a plethora of other off-label uses, is a highly potent, reversible MAO inhibitor. [53] The Food and Drug Administration (FDA) has approved these MAOIs to treat depression: [54]
The most common uses for enzyme inhibitors are as drugs to treat disease. Many of these inhibitors target a human enzyme and aim to correct a pathological condition. For instance, aspirin is a widely used drug that acts as a suicide inhibitor of the cyclooxygenase enzyme. [95]