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Single Injection of rAAV9-CMV-hNAGLUop Gene for Patients With Mucopolysaccharidosis (MPS) IIIB

NeuroGT · 2027
ClinicalTrials.gov · Datasets · License: Public Domain · 2027
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gene-therapygenetic-diseasesmetabolic-diseasesmetabolism
sanfilippo syndrome (mps iii), gene therapy, neurogt, mucopolysaccharidoses, lysosomal storage diseases, congenital, hereditary, and neonatal diseases and abnormalities, metabolism, inborn errors, genetic diseases, inborn, carbohydrate metabolism, inborn errors, mucopolysaccharidosis iii, metabolic diseases, skin and connective tissue diseases
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Record · ID 981099
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