Drug Profile
Research programme: RNA-interference therapeutics - BioMarin
Alternative Names: PRO 052; PRO 055; PRO 105; PRO 135; PRO 289Latest Information Update: 31 May 2018
Price :
$50
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At a glance
- Originator Leiden University
- Developer BioMarin Nederland
- Class Antisense oligonucleotides
- Mechanism of Action Antisense RNA modulators; Dystrophin stimulants
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Orphan Drug Status
Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.
- New Molecular Entity Yes
Highest Development Phases
- Discontinued Duchenne muscular dystrophy; Huntington's disease; Muscular dystrophies; Neuroblastoma; Spinal muscular atrophy