BIOMARIN INTERNATIONAL LIMITED

It is used to treat patients with neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl-peptidase 1 (TPP-1) deficiency. The drug replaces the missing enzyme to slow the progression of the disease.

Dosage form: solution for infusion

ATC: A16AB17

The medicine is used to treat hyperphenylalaninemia (HPA), phenylketonuria (PKU), and the hereditary disorder known as BH4 deficiency, by helping to reduce phenylalanine levels in the blood.

Dosage form: tablets, soluble

ATC: A16AX07

It is used for the treatment of patients suffering from MPS VI (Mucopolysaccharidosis VI), a condition caused by the deficiency of a specific enzyme that leads to the accumulation of certain substances in the tissues.

Dosage form: solution for infusion

ATC: A16AB08

It is a treatment for patients aged 16 years and older suffering from phenylketonuria (PKU), a rare hereditary disorder that causes an accumulation of phenylalanine in the body. The drug helps to reduce the levels of this substance in the blood when it is not possible to control them through diet alone.

Dosage form: solution for injection

ATC: A16AB19

It is used for the treatment of severe hemophilia A in adults who have never had factor VIII inhibitors and who do not have anti-AAV5 vector antibodies.

Dosage form: solution for infusion

ATC: B02

Vimizim is used for the treatment of adults and children with mucopolysaccharidosis Type IVA (MPS IVA), also known as Morquio A syndrome. This medicine replaces a missing enzyme in the body, helping to improve walking and reduce keratan sulfate levels.

Dosage form: solution for infusion, concentrate

ATC: A16AB12

It is used for the treatment of achondroplasia in patients aged 4 months or older whose bones are still growing. It is indicated only in cases of achondroplasia caused by FGFR3 gene mutations confirmed by genetic testing.

Dosage form: solution for injection, powder and solvent

ATC: M05BX07