Research programme: Alpha-L-iduronidase gene therapy - BioMarin

Drug Profile

Research programme: Alpha-L-iduronidase gene therapy - BioMarin

Alternative Names: Human alpha-L-iduronidase gene therapy - BioMarin

Latest Information Update: 28 Aug 2013

Price : $50

At a glance

  • Originator BioMarin Pharmaceutical
  • Class Gene therapies
  • Mechanism of Action Gene transference; Iduronidase replacements
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    No

Highest Development Phases

  • Discontinued Mucopolysaccharidosis I

Most Recent Events

  • 28 Aug 2013 Discontinued - Preclinical for Mucopolysaccharidosis I in USA (Intrathecal)
  • 31 Dec 2010 No development reported - Preclinical for Mucopolysaccharidosis I in USA (Intrathecal)
  • 26 Jul 2006 A preclinical study has been added to the Metabolic Disorders pharmacodynamics section
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