Fig. 4: Clinical assessment of patients over time.
From: Dual-vector rAAVrh8 gene therapy for GM2 gangliosidosis: a phase 1/2 trial
a, CGI severity scores at each time point. Lower scores indicate better neurological status. b,c, Infantile patients’ neurologic exam scores for seizure severity (b) and sucking or swallowing (c). A higher score indicates worsened severity.
