The video assessments focus on several hallmarks of GM1 progression. Caregivers participate in qualitative interviews to provide context for the videos and discuss any changes they observe during the study. Activity videos will be evaluated by expert clinicians. The caregiver interviews and clinician-rated activities will inform the patient-specific disease trajectories for each hallmark.
There is no treatment or intervention associated with this study and data collected will be used as part of the natural history control group for our Gene Therapy trial.