Research programme: spinal muscular atrophy therapeutics - Paratek Pharmaceuticals/Families of SMAAlternative Names: PKT SMA 01; PTK-SMA-01
Latest Information Update: 24 Jun 2009
At a glance
- Originator Families of SMA; Paratek Pharmaceuticals
- Class Small molecules; Tetracyclines
- Mechanism of Action Survival of motor neuron 1 protein stimulants
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.
Highest Development Phases
- Preclinical Spinal muscular atrophy
Most Recent Events
- 17 Jun 2008 Families of Spinal Muscular Atrophy and Paratek Pharmaceuticals have extended their research collaboration for another year
- 03 Apr 2007 Families of Spinal Muscular Atrophy and Paratek Pharmaceuticals have extended their research collaboration
- 24 May 2006 Preclinical trials in Spinal muscular atrophy in USA (unspecified route)