Children with Sanfilippo Type A Given Gene Therapy Showing Signs of Stabilization – Rare Disease Report
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Children with Sanfilippo Type A Given Gene Therapy Showing Signs of Stabilization
Rare Disease Report One year following gene therapy, data from the 3 subjects were compared to data from a natural history study (n=15) and showed a 69.3% reduction in cerebral spinal fluid (CSF) heparan sulfate. The subjects also reported normalization of liver volumes … |
