Liek s účinnou látkou cerliponáza alfa 150 mg, infúzny roztok

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The subject of the contract is Medicine with the active substance cerliponase alfa 150 mg, infusion solution Pharmaceutical form: infusion solution 5 ml/150 mg+5 ml prefilled solution (med.inj.sol.) Administration: intracerebroventricular use The drug cerliponase alfa belongs to the group of medicines known as enzyme replacement therapies. It is used to treat patients with the disease neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase 1 deficiency (TPP1). People with CLN2 disease either do not have the enzyme called TPP1 or have too little of it, which causes the accumulation of substances called lysosomal storage materials. In people with CLN2 disease, these substances accumulate in certain parts of the body, especially the brain. This medicine replaces the missing enzyme called TPPA, thereby minimizing the accumulation of lysosomal storage materials. This medicine works by slowing the progression of the disease. The recommended dose is 300 mg of cerliponase alfa, administered once every two weeks by intracerebroventricular infusion. The subject of the contract is Medicine with the active substance…

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