Clinical and MRI Correlations in GM1 Patients: Finding Biomarkers to Predict Treatment Response with Gene Therapy
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Presenting Author - Maria T. Acosta, MD; Co-Author - Zeynep Vardar, MD, MD; Co-Author - Precilla D'Souza, PNP, DNP; Co-Author - Jean M. Johnston, RN, MS; Co-Author - Anna L. Kuhn, PhD; Co-Author - Audrey E. Thurm, PhD; Co-Author - Cristan Farmer, PhD; Co-Author - Mohammed S. Shazeeb, PhD; Co-Author - Cynthia J. Tifft, MD, PhD;
Meta Tag
Lysosomal Diseases
Methodology
Therapy
Co-Author Zeynep Vardar, MD, MD
Co-Author Precilla D'Souza, PNP, DNP
Co-Author Jean M. Johnston, RN, MS
Co-Author Anna L. Kuhn, PhD
Co-Author Audrey E. Thurm, PhD
Co-Author Cristan Farmer, PhD
Co-Author Mohammed S. Shazeeb, PhD
Co-Author Cynthia J. Tifft, MD, PhD
Presenting Author Maria T. Acosta, MD
Keywords
biomarkers
gene therapy
GM1 gangliosidosis
lysosomal storage disorder
sphingolipid metabolites
clinical endpoints
Vineland Adaptive Behavior Scale
non-invasive MRI techniques
fractional anisotropy
diffusion tensor imaging

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