PPMD’s Preclinical Assessment Laboratory (PAL)

PPMD has been providing support to the Preclinical Assessment Laboratory (PAL) for the last 15 years, by evaluating the therapeutic potential of novel interventions and/or to validate the potential of published interventions in mouse models of Duchenne to help drug developers and PPMD prioritize therapeutic interventions that can potentially move into the clinic.

This program advances drug development by:

  • Providing services to companies who need to repeat or confirm ‘target engagement’ (whether the compound impacts the appropriate pathway) in animal studies in mouse models.
  • Actively engaging with companies whose compounds might target relevant pathways in Duchenne and who are not pursuing Duchenne indications, and seek to interest them to include Duchenne in their portfolio.

Created and led by H. Lee Sweeney, Ph.D., Director of the University of Florida Myology Institute and a member of PPMD’s Scientific Advisory Committee, PAL was established in part to see if there is long-term benefit of interventions in the mouse model of Duchenne which helps to identify best candidates for clinical studies. The assumption is that if an intervention is unable to show sustained benefit in the severe mouse model, then it is unlikely to provide any long-term benefit in people living with Duchenne.

PAL is just one part of PPMD’s Research Strategy. To learn more, click here.