Research programme: lysosomal storage disease therapeutics - Orphazyme ApS
Alternative Names: OR-02; OR-03; Orph-001Latest Information Update: 16 Jul 2016
At a glance
- Originator Orphazyme
- Class Heat shock proteins; Recombinant proteins
- Mechanism of Action Sphingomyelin phosphodiesterase 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
- No development reported Lysosomal storage diseases
Most Recent Events
- 16 Jul 2016 No recent reports of development identified for preclinical development in Lysosomal storage diseases in Denmark (Parenteral)