Intracerebral gene therapy in 4 children with Sanfilippo B syndrome : 5.5 years follow-up results.
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DOI: 10.1089/hum.2021.135
Abstract: We report safety (primary endpoint) and efficacy (secondary endpoint) of novel intracerebral gene therapy at 5.5 years of follow-up in children with Sanfilippo B. An uncontrolled phase 1/2 clinical trial was performed in 4 patients… read more here.
Keywords: children sanfilippo; years follow; gene therapy; intracerebral gene ... See more keywords