Daily Breeze on MSN

South Bay native brings fight for a GM1 gangliosidosis cure to Sacramento

California this weekend will once again recognize a rare and deadly genetic disease that kills most of those affected between the ages of 2 and 4 — in large part because of a local couple who ...
The New England Journal of Medicine

AAV9 Gene Therapy in Type II GM1 Gangliosidosis — A Phase 1–2 Trial

GM1 gangliosidosis, caused by biallelic variants in GLB1, results from deficiency of lysosomal β-galactosidase, which degrades GM1 ganglioside. This fatal neurodegenerative disease currently has no ...
ascopubs.org

Adjuvant Ganglioside GM2-KLH/QS-21 Vaccination Versus Observation After Resection of Primary Tumor > 1.5 mm in Patients With Stage II Melanoma: Results of the EORTC 18961 ...

Prediction of Survival by [18F]Fluorodeoxyglucose Positron Emission Tomography in Patients With Locally Advanced Non–Small-Cell Lung Cancer Undergoing Definitive Chemoradiation Therapy: Results of the ...
ascopubs.org

Survival Impact of Anti-GD2 Antibody Response in a Phase II Ganglioside Vaccine Trial Among Patients With High-Risk Neuroblastoma With Prior Disease Progression

Radical Cystectomy Against Intravesical BCG for High-Risk High-Grade Nonmuscle Invasive Bladder Cancer: Results From the Randomized Controlled BRAVO-Feasibility Study One hundred two patients with ...