Speciality

Genetic Disorders

Disease: MPS VI

Product: Naglazyme® Galsulfase

Indication: Indicated for patients with Mucopolysaccharidosis VI (MPS VI). NAGLAZYME has been shown to improve walking and stair-climbing capacity

Disease: MPS I

Product: Aldurazyme® Laronidase

Indication: Indicated for patients with Hurler and Hurler-Scheie forms of Mucopolysaccharidosis I (MPS I) and for patients with the Scheie form who have moderate to severe symptoms. ALDURAZYME has been shown to improve pulmonary function and walking capacity.

Disease: Gaucher

Product: Cerezyme ® Imiglucerase

Indication: Indicated for long-term enzyme replacement therapy for pediatric and adult patients with a confirmed diagnosis of Type 1 Gaucher disease that results in one or more of the following conditions:• anemia• thrombocytopenia• bone disease

Disease: Pompe

Product: Myozyme ® alglucosidase alfa

Indication: Indicated for use in patients with Pompe disease (GAA deficiency).  MYOZYME has been shown to improve ventilator-free survival in patients with infantile-onset Pompe disease as compared to an untreated historical control, whereas use of MYOZYME in patients with other forms of Pompe disease has not been adequately studied to assure safety and efficacy

Disease: Fabry

Product: Fabarzyme® agalsidase beta

Indication: Indicated for use in patients with Fabry disease.  Fabrazyme reduces globotriaosylceramide (GL-3) deposition in capillary endothelium of the kidney and certain other cell types

Disease: Primary Systemic Carnitine Deficiency.

Product: Carnitor ® Levocarnitine

Indication: Indicated in the treatment of primary systemic carnitine deficiency. Also indicated for acute and chronic treatment of patients with an inborn error of

Disease: Urea Cycle Enzyme Deficiency

Product: Ammonul ® Sodium phenylacetate & Sodium benzoate

Indication: Indicated as adjunctive therapy for the treatment of acute hyperammonemia and associated encephalopathy in patients with deficiencies in enzymes of the urea cycle.

Disease: Urea Cycle Enzyme Deficiency

Product: Ammonaps®Sodium phenylbutyrate

Indication: Indicated as adjunctive therapy in the chronic management of urea cycle disorders, involving deficiencies of carbamylphosphate synthetase, ornithine transcarbamylase or argininosuccinate synthetase. It is indicated in all patients with neonatal-onset presentation (complete enzyme deficiencies, presenting within the first 28 days of life). It is also indicated in patients with late-onset disease (partial enzyme deficiencies, presenting after the first month of life) who have a history of hyperammonaemic encephalopathy

Disease: Hereditary Tyrosinemia Type I 

Product: Cycle Pharma Nitisinone

Indication: Approved for the treatment of   hereditary tyrosinemia type 1 (HT-1) in combination with dietary restriction of tyrosine and phenylalanine