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    • BioMarin Pharmaceutical Inc. drugs

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    8 results

    • brineura

      (cerliponase alfa)
      BioMarin Pharmaceutical Inc.
      Usage: BRINEURA is indicated to slow the loss of ambulation in pediatric patients with neuronal ceroid lipofuscinosis type 2 (CLN2 disease), associated with tripeptidyl peptidase 1 (TPP1) deficiency.

    • kuvan

      (SAPROPTERIN DIHYDROCHLORIDE)
      BioMarin Pharmaceutical Inc.
      Usage: KUVAN® is indicated for reducing blood phenylalanine levels in patients aged one month and older with hyperphenylalaninemia due to tetrahydrobiopterin-responsive Phenylketonuria, to be used alongside a phenylalanine-restricted diet.

    • naglazyme

      (galsulfase)
      BioMarin Pharmaceutical Inc.
      Usage: NAGLAZYME is indicated for patients with Mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy syndrome) and has demonstrated effectiveness in improving walking and stair-climbing capacity.

    • palynziq

      (pegvaliase-pqpz)
      BioMarin Pharmaceutical Inc.
      Usage: Palynziq is indicated for reducing blood phenylalanine levels in adult patients with phenylketonuria (PKU) who have uncontrolled concentrations above 600 micromol/L despite current treatment.

    • roctavian

      (valoctocogene roxaparvovec-rvox)
      BioMarin Pharmaceutical Inc.
      Usage: ROCTAVIAN is indicated for the treatment of adults with severe hemophilia A (congenital factor VIII deficiency with factor VIII activity < 1 IU/dL) who do not have antibodies to adeno-associated virus serotype 5, as verified by an FDA-approved test.

    • vimizim

      (elosulfase alfa)
      BioMarin Pharmaceutical Inc.
      Usage: Vimizim (elosulfase alfa) is indicated for the treatment of Mucopolysaccharidosis type IVA (MPS IVA), also known as Morquio A syndrome.

    • voxzogo 0.4mg

      (vosoritide)
      BioMarin Pharmaceutical Inc.
      Usage: VOXZOGO is indicated for increasing linear growth in pediatric patients with achondroplasia who have open epiphyses. Its approval is based on accelerated approval due to improved annualized growth velocity, with continued approval dependent on further confirmation of clinical benefits.

    • voxzogo 0.4mg - vosoritide

      (Vosoritide)
      Biomarin Pharmaceutical Inc.