How is Myozyme made?
The active substance in Myozyme, alglucosidase alfa, is a copy of human alpha-glucosidase, which is produced by a method known as ‘recombinant DNA technology’: the enzyme is made by a cell that has received a gene (DNA) that makes it able to produce the enzyme.
Are Myozyme and Lumizyme the same?
Myozyme is indicated for infantile-onset Pompe disease; Lumizyme is indicated for patients aged ≥8 years. Although both drugs have been shown to improve patient survival rates, they both also have a boxed warning, because of the possibility of life-threatening allergic reactions.
What does acid alpha-glucosidase do?
Acid alpha-glucosidase is the enzyme responsible for degradation of glycogen polymers to glucose in the acidic milieu of the lysosomes. Cardiac and skeletal muscles are the two major tissues affected by the accumulation of glycogen within the lysosomes.
What is lysosomal alpha-glucosidase?
Acid alpha-glucosidase, also called α-1,4-glucosidase and acid maltase, is an enzyme (EC 3.2. 1.20) that helps to break down glycogen in the lysosome.
How do I administer Myozyme?
It is recommended to start the administration of the diluted solution within three hours. The total time between reconstitution and completion of the infusion should not exceed 24 hours. The recommended dose regimen of Myozyme is 20 mg/kg of body weight administered once every 2 weeks as an intravenous infusion.
How do I infuse Myozyme?
Infusions should be administered incrementally. MYOZYME should be administered at an initial infusion rate of no more than 1 mg/kg/hr. The infusion rate may be increased by 2 mg/kg/hr every 30 minutes, after patient tolerance to the infusion rate is established, until a maximum rate of 7 mg/kg/hr is reached.
When was Lumizyme approved by the FDA?
Lumizyme, a lysosomal glycogen-specific enzyme, was approved by the FDA in 2010 with a REMS to restrict its use to treatment of patients with late (non-infantile) onset Pompe disease who are eight years of age and older.
How do the alpha glucosidase inhibitors work?
How They Work. These drugs block the breakdown of starchy foods such as bread, potatoes, and pasta, and they slow down the absorption of of some sugars, such as table sugar. You take an alpha-glucosidase inhibitor with the first bite of each meal. Most people take a pill three times a day.
What is the function of GAA?
Normal Function The GAA gene provides instructions for producing an enzyme called acid alpha-glucosidase (also known as acid maltase). This enzyme is active in lysosomes, which are structures that serve as recycling centers within cells.
Where is acid alpha-glucosidase produced?
Acid α-glucosidase (GAA) Deficiency (GSDII), or Pompe Disease, is the only glycogen storage disease directly involving abnormal lysosomal metabolism. GAA (also known as acid maltase) is an α-amylase that is localized exclusively to the lysosome.