Making sure every inchstone of progress is measured – Update on The Inchstone Project

Join us for an update on The Inchstone Project which is working to make sure that there are assessments to measure the inchstones, not milestones, of progress our loved ones who are profoundly impacted by rare diseases make. This is important not only for measuring every day progress but also to ensure that as genetic therapies and precision medicines are emerging, there are tools to show improvements in those severely impacted by disorders. If this progress isn’t able to be captured, treatments that help with what is most important to patients and caregivers may not get to market.

Our work is focused on what matters most to families and will touch on the recent DEE Parents Speak survey which heard from nearly 270 families about what their priorities are and what better would look like for their child. (see summary graphic below)