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Early results of gene therapy trial for ‘childhood dementia’ show promise

genetic testing

  • An improvement in neurocognitive assessments compared with natural progression of the disease in one of the children at 18-months post-treatment.
  • Three additional patients are currently within the normal cognitive development range at nine to 18 months post-treatment, but require longer follow-up to assess outcomes.
  • After a median of two years, OTL-201 which was generally well tolerated in all the patients, achieved sustained engraftment in the bone marrow.
  • Higher amounts of the SGSH enzyme were seen than would be normally found in the blood and cerebrospinal fluid of healthy children.

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