Cerliponase alfa
Cerliponase alfa
Cerliponase alfa (pronunciation: ser-li-PO-nase AL-fa) is a medication used for the treatment of a specific type of Batten disease known as CLN2 disease.
Etymology
The name "Cerliponase alfa" is derived from the drug's function. "Cerli-" is a reference to ceroid lipofuscinosis, the medical term for the type of neuronal storage disorder that includes CLN2 disease. "-ponase" refers to the drug's enzyme replacement therapy function, and "alfa" is a common suffix used in biologic medication names to denote a human protein.
Usage
Cerliponase alfa is used to slow the progression of symptoms in patients with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase 1 (TPP1) deficiency. This is a form of Batten disease, a rare and fatal autosomal recessive neurodegenerative disorder.
Mechanism of Action
Cerliponase alfa works by replacing the TPP1 enzyme, which is missing or not working properly in people with CLN2 disease. This enzyme is necessary for normal cell function, and its absence leads to a buildup of proteins and lipids in the cells, causing the symptoms of the disease.
Related Terms
- Enzyme replacement therapy: A medical treatment which replaces an enzyme that is deficient or absent in the body.
- Neuronal ceroid lipofuscinosis: A group of inherited neurodegenerative disorders characterized by the accumulation of lipofuscin in the body's tissues.
- Batten disease: A rare, fatal autosomal recessive neurodegenerative disorder that begins in childhood.
External links
- Medical encyclopedia article on Cerliponase alfa
- Wikipedia's article - Cerliponase alfa
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