Trial sites and patients
Defined patient cohorts and experienced trial sites and PIs are essential requirements for any trial. TREAT-NMD has created a global registry of trial sites with key data on their expertise, and global registries of patients with DMD and SMA* containing the necessary genetic and clinical information to allow filtering by inclusion criteria. Together these two services form an immensely valuable tool that substantially lowers the hurdles to getting a trial off the ground.
*registries for other conditions in preparation