BioMarin is providing innovative therapeutics to patients with serious unmet medical needs. All of their commercialized products are for rare genetic disorders.
Ikris Pharma Network is authorized by BioMarin to provide access to their medicines to Indian patients through Named-Patient Import.
Vimizim® (elosulfase alfa) is the only enzyme replacement therapy (ERT) to address the cause of Morquio A (MPS IVA). People born with Morquio A have a gene that does not work correctly. This gene results in missing or low levels of the N-acetylgalactosamine-6 sulfatase (GALNS) enzyme that breaks down certain complex carbohydrates known as glycosaminoglycans (GAGs). Without sufficient quantities of GALNS, the normal breakdown of GAG is incomplete or blocked and it accumulates in the lysosomes of the cell.
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Naglazyme® (galsulfase) is an enzyme replacement therapy for the treatment of Mucopolysaccharidosis VI (MPS VI), an inherited life-threatening lysosomal storage disorder caused by a deficiency of the lysosomal enzyme N-acetylgalactosamine 4-sulfatase, an enzyme normally required for the breakdown of certain complex carbohydrates known as as glycosaminoglycans (GAGs).
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Kuvan is the first and only FDA-approved medication for PKU to reduce blood Phe levels in patients with hyperphenylalaninemia (HPA) due to tetrahydrobiopterin (BH4-) responsive PKU. Kuvan is a pharmaceutical formulation of BH4, the natural cofactor for the PAH enzyme, which stimulates activity of the residual PAH enzyme to metabolize Phe into tyrosine. Kuvan is to be used in conjunction with a Phe-restricted diet.
Kuvan at a Glance
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PALYNZIQ® (pegvaliase-pqpz) Injection is the first FDA-approved enzyme substitution therapy designed to address the underlying cause of phenylketonuria (PKU), a rare and genetic brain-threatening condition marked by an inability to break down phenylalanine (Phe). PALYNZIQ is a once-daily self-administered therapy, clinically proven to reduce blood Phe levels in adults with PKU who have uncontrolled blood Phe levels on existing management.
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Brineura® (cerliponase alfa) is the first approved treatment for any form of Batten disease, and is indicated to slow the loss of ambulation in symptomatic pediatric patients 3 years of age and older with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase 1 (TPP1) deficiency.i CLN2 disease is an ultra-rare and rapidly progressing brain disorder that affects an approximately estimated 20 children born in the United States each year– less than one in a million Americans
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