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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]
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]
In the middle of the 20th century the principal treatment for some of the amino acid disorders was restriction of dietary protein and all other care was simply management of complications. In the past twenty years, new medications, enzyme replacement, gene therapy, and organ transplantation have become available and beneficial for many ...
It is an X-linked recessive disorder that results in defective glucose-6-phosphate dehydrogenase enzyme. [1] Glucose-6-phosphate dehydrogenase is an enzyme which protects red blood cells, which carry oxygen from the lungs to tissues throughout the body. A defect of the enzyme results in the premature breakdown of red blood cells.
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 ...
Phosphodiesterase-5. A phosphodiesterase inhibitor is a drug that blocks one or more of the five subtypes of the enzyme phosphodiesterase (PDE), thereby preventing the inactivation of the intracellular second messengers, cyclic adenosine monophosphate (cAMP) and cyclic guanosine monophosphate (cGMP) by the respective PDE subtype(s).
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] They help the person digest fats, starches, and proteins. [5] Pancreatic enzymes have been used as medications since at least the 1800s. [7]
Thus far, Periostat (active ingredient is doxycycline hyclate) is the only MMP inhibitor that has been approved by the U.S. Food and Drug Administration (FDA). It is used for the treatment of periodontitis. Other MMP inhibitors have exhibited serious side effects during preclinical trials. These side effects are caused by insufficient selectivity.