Drug Profile
Research programme: Alpha-L-iduronidase gene therapy - BioMarin
Alternative Names: Human alpha-L-iduronidase gene therapy - BioMarinLatest Information Update: 28 Aug 2013
Price :
$50
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At a glance
- Originator BioMarin Pharmaceutical
- Class Gene therapies
- Mechanism of Action Gene transference; Iduronidase replacements
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Orphan Drug Status
Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.
Highest Development Phases
- Discontinued Mucopolysaccharidosis I