Last Updated: 09/26/2026
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Found 8 publications
AAV9 Gene Therapy in Type II GM1 Gangliosidosis - A Phase 1-2 Trial.
Journal: The New England journal of medicine
Published: February 10, 2026
AAV9 Gene Therapy in GM1 Gangliosidosis Type II: A Phase 1/2 Trial.
Journal: medRxiv : the preprint server for health sciences
Published: August 06, 2025
Brain Age Prediction in Type II GM1 Gangliosidosis.
Journal: medRxiv : the preprint server for health sciences
Published: May 02, 2025
Differential tractography: an imaging marker for tissue degeneration in neurodegenerative diseases.
Journal: Brain communications
Published: September 11, 2024
Risks and benefits of anesthesia for combined pediatric procedures in the NIH undiagnosed diseases program.
Journal: Molecular genetics and metabolism
Published: July 07, 2023
Anesthesia outcomes in lysosomal disorders: CLN3 and GM1 gangliosidosis.
Journal: American journal of medical genetics. Part A
Published: August 22, 2022
Showing 1-8 of 8
Last Updated: 09/26/2026