Research programme: rare disease therapies - Shire/Telethon
Latest Information Update: 16 Jul 2016
At a glance
- Originator Fondazione Telethon; Shire
- Mechanism of Action Undefined mechanism
Orphan Drug Status
Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.
On Fast track
Fast track status is assigned by the US FDA so therapies with the potential to address unmet needs can move faster through development.
- New Molecular Entity Yes
Highest Development Phases
- No development reported Lysosomal storage diseases; Neurodegenerative disorders
Most Recent Events
- 16 Jul 2016 No recent reports of development identified for research development in Lysosomal storage diseases in Italy
- 16 Jul 2016 No recent reports of development identified for research development in Neurodegenerative-disorders in Italy
- 24 Oct 2012 Early research in Neurodegenerative disorders in Italy (unspecified route)