Unravelling the Latest Research in Disease Pathology to Explore Future Considerations in Drug Development

Time: 11:30 am - 2:30 pm
day: Pre-Conference Workshop Day


With both Rett and Fragile X Syndrome having experienced recent clinical failures, there remains a need for greater disease understanding. Despite differing severity of clinical presentation and molecular etiology, the impairment of synaptic function as well as plasticity is seen in both diseases. Here, we will deep dive into recent advances in disease understanding to encourage successful and predictive results in future clinical studies.

This workshop will cover:

  • Discovering what the last 12 months has brought for our understanding of Rett and Fragile X Syndrome
  • Exploring essential considerations from an enhanced disease understanding: outcome measures and trial design
  • Identifying what gaps in research need to be explored in the future to achieve clinical success