In May 2020, Aparito presented a poster at the 10th European Conference on Rare Diseases (ECRD).

The poster explored how video-based Patient-Reported Outcomes (PROs) have been used to record and measure disease post gene therapy in patients with Sanfilippo Disease.

Interim conclusions include:

  • Qualitative interviews provide key information for interpreting videos
  • Video and context allows capture of subtle but meaningful incremental changes
  • The method is feasible for the longer-term monitoring of real-world functioning and patient status