Cristan Farmer

This phase 1/2 trial evaluates AAV9 gene therapy for GM1 gangliosidosis Type II, showing safety and some biochemical and neuroimaging improvements.

This paper develops meaningful score differences (MSDs) for the Bayley-4 and Vineland-3 in Angelman syndrome using a Delphi panel, finding that MSD thresholds vary with baseline functioning severity.

The paper investigates spindle chirp and other sleep oscillatory features in young children with and without autism, finding significant differences in chirp negativity and coupling angles.

This study presents a 10-year prospective analysis of type II GM1 gangliosidosis, detailing disease progression and phenotypic features in 41 patients.